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43 results in Model Systems

Ai14 mouse Model System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Ai14 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. The att site flanked neo selection cassette has been removed in this strain. Show Experiments (1) Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
Ai14 mouse (congenic) Model System - [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Ai14 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. The att site flanked neo selection cassette has been removed in this strain. Show Experiments (4) Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea Independent validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Repeat experiment of independent validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Independent validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
Ai14 mouse primary fibroblasts Model System - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Primary fibroblasts from Ai14 mice Show Experiments (1) Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea
Ai9 mouse Model System - [In Vivo] [Small Animal Testing Center (SATC), AAV tropism, Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Show Experiments (11) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Testing AAV5 for activation of tdTomato in mouse airway Cre Recombinase dose escalation study in Ai9 mice Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector. Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain FUS (focused ultrasound) array validation in Ai9 mice Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells AAV Tropism project
Ai9 mouse (BCM) Model System - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Show Experiments (2) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Ai9 mouse immortalized fibroblasts Model System - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Immortalized fibroblasts made from Ai9 (B6.Cg-Gt(ROSA)26Sor^tm9(CAG-tdTomato)Hze/J) mice Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Ai9-SauSpyCas9 mouse Model System - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Ai9-SauSpyCas9 is a version of Ai9 that has a single guide RNA sites on both sides of the STOP cassette. This is a Cre reporter allele that has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Ai9 mice express robust tdTomato fluorescence following Cre-mediated recombination. Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
B6 Model System - [In Vivo] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System C57BL/6J WT mouse Show Experiments (1) Independent validation of Chaikof delivery platform using virus-like particle (VLP) delivery to the mouse liver
BALB/c mouse Model System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System BALB/cJ is a commonly used inbred. Key traits include a susceptibility to developing the demyelinating disease upon infection with Theiler's murine encephalomyelitis virus. The BALB/cJ substrain is susceptible to Listeria, all species of Leishmania, and several species of Trypanosoma, but is resistant to experimental allergic orchitis (EAO). Show Experiments (1) Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
C57BL/6 mouse (Asokan study) Model System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Show Experiments (2) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
C57BL/6J mouse Model System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System C57BL/6J WT mouse Show Experiments (3) Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice Pcsk9 adenine base editor efficiency in liver and nonliver tissue On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver
CD4/CD8 Human Primary T cell Model System - [In Vitro] [Biological Systems] [Human] Matched Fields: category : Model System CD4/CD8 Human Primary T cell Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
GER10 Heterozygous Blastocyst Model System - [In Vitro] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Blastocyst derived from GER10 heterozygous mice Show Experiments (1) Validating Gene Editing Reporter 10 (GER10) Mouse Model in Heterozygous Blastocysts
GER12 Heterozygous Blastocyst Model System - [In Vitro] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Blastocyst derived from GER12 heterozygous mice Show Experiments (1) Validating Gene Editing Reporter 12 (GER12) Mouse Model in Heterozygous Blastocysts
GER14 Heterozygous Blastocyst Model System - [In Vitro] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Blastocyst derived from GER14 heterozygous mice Show Experiments (1) Validating Gene Editing Reporter 14 (GER14) Mouse Model in Heterozygous Blastocysts
GER18 Heterozygous Blastocyst Model System - [In Vitro] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Blastocyst derived from GER18 heterozygous mice Show Experiments (1) Validating Gene Editing Reporter 18 (GER18) Mouse Model in Heterozygous Blastocysts
GER19 Heterozygous Blastocyst Model System - [In Vitro] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Blastocyst derived from GER19 heterozygous mice Show Experiments (1) Validating Gene Editing Reporter 19 (GER19) Mouse Model in Heterozygous Blastocysts
HEK-293-TgEF1a-eGFP-BSD Model System - [In Vitro] [Genome Editors] [Human] Matched Fields: category : Model System HEK293 cells with lentiviral insertion of EF1a promoter driving expression of eGFP and SV40 promoter driving expression of BSD. HEK293 is an epithelial-like cell that was isolated from the kidney of a patient. Show Experiments (1) Testing newly discovered Biggie Phage editors in human cells
HEK-293T with Ai9 transient reporter assay Model System - [In Vitro] [Delivery Systems Initiative] [Human] Matched Fields: category : Model System HEK-293T cells transfected with an Ai9 inducible transgene reporter plasmid used to test gene editing activity by fluorescence. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient. Show Experiments (1) Testing AAV5 for activation of tdTomato in HEK293T cells
HEK-293T-disrupted_GFP with MCV-mcherry-Puro Model System - [In Vitro] [Delivery Systems Initiative] [Human] Matched Fields: category : Model System HEK293T cells with an integrated reporter for TLR-MCV1 reporter editing. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient. Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA to activate the TLR reporter in human cells
HEK-293T-disrupted_GFP-mcherry-Puro Model System - [In Vitro] [Delivery Systems Initiative] [Human] Matched Fields: category : Model System HEK293T cells with an integrated reporter for TLR1 reporter editing. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient. Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA to activate the TLR1 reporter in human cells
HeLa Model System - [In Vitro] [Collaborative Opportunity Fund] [Human] Matched Fields: category : Model System HeLa cells were isolated in 1951 from a cervical carcinoma; Epithelial cell; Uterus; Cervix Show Experiments (1) Inhibitory effect of Cas9 RNP alone on S10- or FSDS315-mediated GFP protein delivery to HeLa cells
Hepa1-6 Model System - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Stable mouse cell line of liver epithelial cells. Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA in mouse Hepa 1-6 cells
Human kidney organoid (ESC-derived) Model System - [In Vitro] [Biological Effects Initiative] [Human] Matched Fields: category : Model System Kidney organoid derived from human female H9 embryonic stem cells Show Experiments (1) Biomarker assays to evaluate toxicity and inflammation induced by AAVs in ES cell derived kidney organoids.
Human kidney organoid (iPSC-derived) Model System - [In Vitro] [Biological Effects Initiative] [Human] Matched Fields: category : Model System Kidney organoid derived from human male BJFF iPS cells Show Experiments (2) AAV2/2 transduction efficiencies in kidney organoids cultured under flow determined by FACS Biomarker assays to evaluate toxicity induced by AAVs in iPS cell derived kidney organoids.
Human kidney organoid (stem cell-derived) Model System - [In Vitro] [Biological Effects Initiative] [Human] Matched Fields: category : Model System Kidney organoid derived from human female H9 ES or BJFF iPS cells Show Experiments (4) AAV tropism in kidney organoids derived from human pluripotent stem cells. AAV tropism in kidney organoids cultured under flow Delivery of saCas9 and gRNAs to nephron epithelia by AAV2 in kidney organoids. Evaluation of DNA damage, cellular toxicity and inflammatory markers following saCas9 and gRNAs delivery by AAV2 in kidney organoids.
K562 Model System [Human] Matched Fields: category : Model System
Liver On Chip (LoC) Model System - [In Vitro] [Collaborative Opportunity Fund] [Human] Matched Fields: category : Model System organoids derived from primary human hepatocytes Show Experiments (3) Testing different ratios of Lipofectamine-RNPs after 24 hours for determination of positive control Imaging quantification of transfection efficiency with varying dosages of nanoparticles encapsulated with Cas9/sgRNA RNP on the liver-on-chip model system Quantification of transfection efficiency by flow cytometry with varying dosages of nanoparticles encapsulated with Cas9/sgRNA RNP on liver-on-chip model system
Macaca mulatta (Rhesus Macaque) Model System - [In Vivo] [Collaborative Opportunity Fund] [Rhesus macaque] Matched Fields: category : Model System Show Experiments (1) Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
Mouse Embryonic Fibroblasts Model System - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Primary cell line Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA in mTmG mouse embryonic fibroblasts
NK Model System - [In Vitro] [Delivery Systems Initiative] [Human] Matched Fields: category : Model System Natural Killer cells. White blood cells; Show Experiments (1) Gene editing in vitro by various peptide variants delivering Cas12a in NK cells.
Neuro 2A Model System - [In Vitro] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System Neuro-2a cells are mouse neuroblasts with neuronal and amoeboid stem cell morphology isolated from brain tissue. Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA in mouse Neuro 2A cells
Primary Airway Epithelia (Human) Model System - [In Vitro] [Collaborative Opportunity Fund, Delivery Systems Initiative] [Human] Matched Fields: category : Model System Primary airway epithelia from non-CF donors Show Experiments (4) Peptide Shuttle optimization to deliver Cas9 RNP to human airway epithelia cells Peptide Shuttle optimization to deliver Cas12a RNP to human airway epithelia cells Gene editing in vitro by various peptide variants delivering Cas RNPs to primary Human airway epithelia cells. Delivery of Cas9 RNP using Shuttle peptide candidates to human airway epithelial cells cultured at the air liquid interface targeting CFTR locus
Primary Airway Epithelia (Human) Model System - [In Vitro] [Collaborative Opportunity Fund] [Human] Matched Fields: category : Model System Primary Airway Epithelia cultured at the air liquid interface Show Experiments (1) Delivery of ABE8e-Cas9 RNP using Shuttle peptide candidates to human airway epithelial cells cultured at the air liquid interface targeting B2M locus
Primary Airway Epithelia (Rhesus Macaque) Model System - [In Vitro] [Collaborative Opportunity Fund] [Rhesus macaque] Matched Fields: category : Model System Rhesus macaque airway epithelial cells Show Experiments (1) Delivery of ABE8e-Cas9 RNP using Shuttle peptide candidates to rhesus monkey airway epithelial cells cultured at the air liquid interface targeting CCR5 locus.
Primary skin fibroblasts (Rhesus Macaque) Model System - [In Vitro] [Collaborative Opportunity Fund] [Rhesus macaque] Matched Fields: category : Model System Rhesus macaque primary skin fibroblasts Show Experiments (2) Rhesus macaque CCR5 gRNA screening using ABE8e-Cas9 Rhesus macaque CCR5 gRNA screening using ABE8e-Cas12a
TLR-2 mouse heterozygous blastocyst Model System - [In Vitro] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Blastocyst derived from TLR2 heterozygous mice Show Experiments (2) Validating Traffic Light Reporter 2 (TLR2) Mouse Model in Heterozygous Blastocysts using SpCas9 Validating Traffic Light Reporter 2 (TLR2) Mouse Model in Heterozygous Blastocysts using SaCas9
TLR-2 mouse homozygous blastocyst Model System - [In Vitro] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Blastocyst derived from TLR2 homozygous mice Show Experiments (1) Validating Traffic Light Reporter 2 (TLR2) Mouse Model in Homozygous Blastocysts using SpCas9
TLR-7 mouse heterozygous blastocyst Model System - [In Vitro] [Small Animal Testing Center (SATC)] [Mouse] Matched Fields: category : Model System Blastocyst derived from TLR7 heterozygous mice Show Experiments (2) Validating Traffic Light Reporter 7 (TLR7) Mouse Model for NHEJ in Heterozygous Blastocysts Validating Traffic Light Reporter 7 (TLR7) Mouse Model for HDR in Heterozygous Blastocysts
TLR-MCV1 mouse Model System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System TLR-MCV1 transgene knocked into Rosa26 locus Show Experiments (1) Delivery of RNP containing chemically modified crRNA C20 with chemically modified tracrRNA T2-PS to determine the RNP distribution in TLR-MCV mouse brain
human kidney organoid with Ai9 (tdTomato) fluorescence-on reporter Model System - [In Vitro] [Collaborative Opportunity Fund] [Human] Matched Fields: category : Model System Kidney organoids were derived from a human iPS cell line with Ai9 (tdTomato) fluorescence-on reporter knocked into the AAVS1 safe harbor locus. Show Experiments (1) Podocyte-specific gene editing in human kidney organoids
mTmG mouse Model System - [In Vivo] [Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System ROSAmT/mG is a cell membrane-targeted, two-color fluorescent Cre-reporter allele. Prior to Cre recombination, cell membrane-localized tdTomato (mT) fluorescence expression is widespread in cells/tissues. Cre recombinase expressing cells (and future cell lineages derived from these cells) have cell membrane-localized EGFP (mG) fluorescence expression replacing the red fluorescence Show Experiments (1) Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
mTmG mouse (congenic) Model System - [In Vivo] [Small Animal Testing Center (SATC), Delivery Systems Initiative] [Mouse] Matched Fields: category : Model System mTmG is a double-fluorescent reporter transgenic mouse which expresses membrane-targeted tdTomato flanked by loxP sequences, followed by membrane-targeted GFP. After genomic cleavage by Cas9 at two sites, or Cre recombinase between loxP sites, tdTomato expression is lost and GFP is expressed. Show Experiments (6) Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA using the S10 shuttle peptide to activate the mTmG reporter in mouse brain Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Independent validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides Testing preparation for independent validation at The Jackson Laboratory Small Animal Testing Center

43 results in Model Systems

Type	Organism	Subtype	Name	Description	Source	View Associated..
Animal	Mouse		Ai14 mouse	Ai14 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. The att site flanked neo selection cassette has been removed in this strain.	The Jackson Laboratory	Show Experiments (1) Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain.
Animal	Mouse		Ai14 mouse (congenic)	Ai14 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. The att site flanked neo selection cassette has been removed in this strain.	The Jackson Laboratory	Show Experiments (4) Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in adult mouse cochlea Independent validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Repeat experiment of independent validation of Chen delivery platform using LNPs to deliver CRISPR/Cas9 to mouse inner ear Independent validation of Gong delivery platform using RNP-loaded nanocages to deliver CRISPR/Cas9 to mouse brain
Cell	Mouse	Primary cells	Ai14 mouse primary fibroblasts	Primary fibroblasts from Ai14 mice		Show Experiments (1) Testing new LNPs (lipid nanoparticles) for delivery of Cas9 mRNA/sgRNA in primary fibroblast cells from adult Ai14 mouse cochlea
Animal	Mouse		Ai9 mouse	Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.	The Jackson Laboratory	Show Experiments (11) Testing gRNA sequence and gRNA scaffold modified in Ai9 mice. Testing AAV5 for activation of tdTomato in mouse airway Cre Recombinase dose escalation study in Ai9 mice Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector. Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain FUS (focused ultrasound) array validation in Ai9 mice Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells AAV Tropism project
Animal	Mouse		Ai9 mouse (BCM)	Ai9 mouse has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus.	Baylor College of Medicine	Show Experiments (2) Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Cell	Mouse	Immortalized Fibroblast (unspecified)	Ai9 mouse immortalized fibroblasts	Immortalized fibroblasts made from Ai9 (B6.Cg-Gt(ROSA)26Sor^tm9(CAG-tdTomato)Hze/J) mice		Show Experiments (1) Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro
Animal	Mouse		Ai9-SauSpyCas9 mouse	Ai9-SauSpyCas9 is a version of Ai9 that has a single guide RNA sites on both sides of the STOP cassette. This is a Cre reporter allele that has a loxP-flanked STOP cassette preventing transcription of a CAG promoter-driven red fluorescent protein variant (tdTomato) - all inserted into the Gt(ROSA)26Sor locus. Ai9 mice express robust tdTomato fluorescence following Cre-mediated recombination.	Baylor College of Medicine	Show Experiments (1) Independent validation for Gao Delivery Team: Testing ssAAV5 delivered intratracheally for editing activity in lung epithelia in Ai9 mice
Animal	Mouse		B6	C57BL/6J WT mouse	Baylor College of Medicine	Show Experiments (1) Independent validation of Chaikof delivery platform using virus-like particle (VLP) delivery to the mouse liver
Animal	Mouse		BALB/c mouse	BALB/cJ is a commonly used inbred. Key traits include a susceptibility to developing the demyelinating disease upon infection with Theiler's murine encephalomyelitis virus. The BALB/cJ substrain is susceptible to Listeria, all species of Leishmania, and several species of Trypanosoma, but is resistant to experimental allergic orchitis (EAO).	The Jackson Laboratory	Show Experiments (1) Testing Shuttle Peptides ability to deliver GFP-NLS to airway epithelia.
Animal	Mouse		C57BL/6 mouse (Asokan study)		Unspecified	Show Experiments (2) Testing virus region 4 (VR4) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice. Testing virus region 8 (VR8) mutant cross-species compatible Adeno Associated Viruses (ccAAVs) in mice.
Animal	Mouse		C57BL/6J mouse	C57BL/6J WT mouse	The Jackson Laboratory	Show Experiments (3) Testing new LNPs (lipid nanoparticles) for delivery of Fluc mRNA in adult mice Pcsk9 adenine base editor efficiency in liver and nonliver tissue On-target editing compared to 14 circle-seq nominated off-target sites of adenine base editor delivered by BE-eVLP vs AAV in the C57BL/6 liver
Cell	Human	Primary cells	CD4/CD8 Human Primary T cell	CD4/CD8 Human Primary T cell	Key Biologicals	Show Experiments (1) A novel human T cell platform to define biological adverse effects of genome editing
Cell	Mouse	Primary cells	GER10 Heterozygous Blastocyst	Blastocyst derived from GER10 heterozygous mice	The Jackson Laboratory	Show Experiments (1) Validating Gene Editing Reporter 10 (GER10) Mouse Model in Heterozygous Blastocysts
Cell	Mouse	Primary cells	GER12 Heterozygous Blastocyst	Blastocyst derived from GER12 heterozygous mice	The Jackson Laboratory	Show Experiments (1) Validating Gene Editing Reporter 12 (GER12) Mouse Model in Heterozygous Blastocysts
Cell	Mouse	Primary cells	GER14 Heterozygous Blastocyst	Blastocyst derived from GER14 heterozygous mice	The Jackson Laboratory	Show Experiments (1) Validating Gene Editing Reporter 14 (GER14) Mouse Model in Heterozygous Blastocysts
Cell	Mouse	Primary cells	GER18 Heterozygous Blastocyst	Blastocyst derived from GER18 heterozygous mice	The Jackson Laboratory	Show Experiments (1) Validating Gene Editing Reporter 18 (GER18) Mouse Model in Heterozygous Blastocysts
Cell	Mouse	Primary cells	GER19 Heterozygous Blastocyst	Blastocyst derived from GER19 heterozygous mice	The Jackson Laboratory	Show Experiments (1) Validating Gene Editing Reporter 19 (GER19) Mouse Model in Heterozygous Blastocysts
Cell	Human	Immortalized	HEK-293-TgEF1a-eGFP-BSD	HEK293 cells with lentiviral insertion of EF1a promoter driving expression of eGFP and SV40 promoter driving expression of BSD. HEK293 is an epithelial-like cell that was isolated from the kidney of a patient.		Show Experiments (1) Testing newly discovered Biggie Phage editors in human cells
Cell	Human	Immortalized	HEK-293T with Ai9 transient reporter assay	HEK-293T cells transfected with an Ai9 inducible transgene reporter plasmid used to test gene editing activity by fluorescence. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient.		Show Experiments (1) Testing AAV5 for activation of tdTomato in HEK293T cells
Cell	Human	Immortalized	HEK-293T-disrupted_GFP with MCV-mcherry-Puro	HEK293T cells with an integrated reporter for TLR-MCV1 reporter editing. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient.		Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA to activate the TLR reporter in human cells
Cell	Human	Immortalized	HEK-293T-disrupted_GFP-mcherry-Puro	HEK293T cells with an integrated reporter for TLR1 reporter editing. HEK293T is an epithelial-like cell that was isolated from the kidney of a patient.		Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA to activate the TLR1 reporter in human cells
Cell	Human		HeLa	HeLa cells were isolated in 1951 from a cervical carcinoma; Epithelial cell; Uterus; Cervix	ATCC	Show Experiments (1) Inhibitory effect of Cas9 RNP alone on S10- or FSDS315-mediated GFP protein delivery to HeLa cells
Cell	Mouse	Hepatoma	Hepa1-6	Stable mouse cell line of liver epithelial cells.	ATCC	Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA in mouse Hepa 1-6 cells
Organoid	Human		Human kidney organoid (ESC-derived)	Kidney organoid derived from human female H9 embryonic stem cells	Morizane lab	Show Experiments (1) Biomarker assays to evaluate toxicity and inflammation induced by AAVs in ES cell derived kidney organoids.
Organoid	Human		Human kidney organoid (iPSC-derived)	Kidney organoid derived from human male BJFF iPS cells	Morizane lab	Show Experiments (2) AAV2/2 transduction efficiencies in kidney organoids cultured under flow determined by FACS Biomarker assays to evaluate toxicity induced by AAVs in iPS cell derived kidney organoids.
Organoid	Human		Human kidney organoid (stem cell-derived)	Kidney organoid derived from human female H9 ES or BJFF iPS cells	Morizane lab	Show Experiments (4) AAV tropism in kidney organoids derived from human pluripotent stem cells. AAV tropism in kidney organoids cultured under flow Delivery of saCas9 and gRNAs to nephron epithelia by AAV2 in kidney organoids. Evaluation of DNA damage, cellular toxicity and inflammatory markers following saCas9 and gRNAs delivery by AAV2 in kidney organoids.
Cell	Human		K562
Organoid	Human		Liver On Chip (LoC)	organoids derived from primary human hepatocytes		Show Experiments (3) Testing different ratios of Lipofectamine-RNPs after 24 hours for determination of positive control Imaging quantification of transfection efficiency with varying dosages of nanoparticles encapsulated with Cas9/sgRNA RNP on the liver-on-chip model system Quantification of transfection efficiency by flow cytometry with varying dosages of nanoparticles encapsulated with Cas9/sgRNA RNP on liver-on-chip model system
Animal	Rhesus macaque		Macaca mulatta (Rhesus Macaque)		Tarantal Lab	Show Experiments (1) Amphiphilic Peptides Deliver Base Editor RNPs to Rhesus Monkey Airway
Cell	Mouse	Primary cells	Mouse Embryonic Fibroblasts	Primary cell line	In-house: https://jacks-lab.mit.edu/protocols/making_mefs	Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA in mTmG mouse embryonic fibroblasts
Cell	Human	Primary cells	NK	Natural Killer cells. White blood cells;	GreenCross LabCell	Show Experiments (1) Gene editing in vitro by various peptide variants delivering Cas12a in NK cells.
Cell	Mouse	Neuroblastoma	Neuro 2A	Neuro-2a cells are mouse neuroblasts with neuronal and amoeboid stem cell morphology isolated from brain tissue.	ATCC	Show Experiments (1) Testing newly chemically modified crRNA and tracrRNA in mouse Neuro 2A cells
Cell	Human	Primary cells	Primary Airway Epithelia (Human)	Primary airway epithelia from non-CF donors	University of Iowa	Show Experiments (4) Peptide Shuttle optimization to deliver Cas9 RNP to human airway epithelia cells Peptide Shuttle optimization to deliver Cas12a RNP to human airway epithelia cells Gene editing in vitro by various peptide variants delivering Cas RNPs to primary Human airway epithelia cells. Delivery of Cas9 RNP using Shuttle peptide candidates to human airway epithelial cells cultured at the air liquid interface targeting CFTR locus
Cell	Human	Primary cells	Primary Airway Epithelia (Human)	Primary Airway Epithelia cultured at the air liquid interface	Cystic Fibrosis Foundation or McGill University	Show Experiments (1) Delivery of ABE8e-Cas9 RNP using Shuttle peptide candidates to human airway epithelial cells cultured at the air liquid interface targeting B2M locus
Cell	Rhesus macaque	Primary cells	Primary Airway Epithelia (Rhesus Macaque)	Rhesus macaque airway epithelial cells	Tarantal Lab	Show Experiments (1) Delivery of ABE8e-Cas9 RNP using Shuttle peptide candidates to rhesus monkey airway epithelial cells cultured at the air liquid interface targeting CCR5 locus.
Cell	Rhesus macaque	Primary cells	Primary skin fibroblasts (Rhesus Macaque)	Rhesus macaque primary skin fibroblasts	Tarantal Lab	Show Experiments (2) Rhesus macaque CCR5 gRNA screening using ABE8e-Cas9 Rhesus macaque CCR5 gRNA screening using ABE8e-Cas12a
Cell	Mouse	Primary cells	TLR-2 mouse heterozygous blastocyst	Blastocyst derived from TLR2 heterozygous mice	The Jackson Laboratory	Show Experiments (2) Validating Traffic Light Reporter 2 (TLR2) Mouse Model in Heterozygous Blastocysts using SpCas9 Validating Traffic Light Reporter 2 (TLR2) Mouse Model in Heterozygous Blastocysts using SaCas9
Cell	Mouse	Primary cells	TLR-2 mouse homozygous blastocyst	Blastocyst derived from TLR2 homozygous mice	The Jackson Laboratory	Show Experiments (1) Validating Traffic Light Reporter 2 (TLR2) Mouse Model in Homozygous Blastocysts using SpCas9
Cell	Mouse	Primary cells	TLR-7 mouse heterozygous blastocyst	Blastocyst derived from TLR7 heterozygous mice	The Jackson Laboratory	Show Experiments (2) Validating Traffic Light Reporter 7 (TLR7) Mouse Model for NHEJ in Heterozygous Blastocysts Validating Traffic Light Reporter 7 (TLR7) Mouse Model for HDR in Heterozygous Blastocysts
Animal	Mouse		TLR-MCV1 mouse	TLR-MCV1 transgene knocked into Rosa26 locus		Show Experiments (1) Delivery of RNP containing chemically modified crRNA C20 with chemically modified tracrRNA T2-PS to determine the RNP distribution in TLR-MCV mouse brain
Organoid	Human		human kidney organoid with Ai9 (tdTomato) fluorescence-on reporter	Kidney organoids were derived from a human iPS cell line with Ai9 (tdTomato) fluorescence-on reporter knocked into the AAVS1 safe harbor locus.	Beno Freedman Lab	Show Experiments (1) Podocyte-specific gene editing in human kidney organoids
Animal	Mouse		mTmG mouse	ROSAmT/mG is a cell membrane-targeted, two-color fluorescent Cre-reporter allele. Prior to Cre recombination, cell membrane-localized tdTomato (mT) fluorescence expression is widespread in cells/tissues. Cre recombinase expressing cells (and future cell lineages derived from these cells) have cell membrane-localized EGFP (mG) fluorescence expression replacing the red fluorescence	The Jackson Laboratory	Show Experiments (1) Shuttle peptides enable in vivo gene editing with Cas9 and Cas12a RNP in mouse airway epithelia
Animal	Mouse		mTmG mouse (congenic)	mTmG is a double-fluorescent reporter transgenic mouse which expresses membrane-targeted tdTomato flanked by loxP sequences, followed by membrane-targeted GFP. After genomic cleavage by Cas9 at two sites, or Cre recombinase between loxP sites, tdTomato expression is lost and GFP is expressed.	The Jackson Laboratory	Show Experiments (6) Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA using the S10 shuttle peptide to activate the mTmG reporter in mouse brain Delivery of unmodified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Delivery of chemically modified, phosphorothioate (PS)-stabilized crRNA with chemically modified, extended PS-stabilized tracrRNA to activate the mTmG reporter in mouse brain Independent validation for McCray Delivery Team: Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides Testing preparation for independent validation at The Jackson Laboratory Small Animal Testing Center

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43 results in Model Systems

43 results in Model Systems