Delivery Systems Projects

  • The initiative goal is to support the development and evaluation of innovative approaches to deliver genome editing machinery into somatic cells, with the ultimate goal of accelerating the development of genome editing therapeutics to treat human disease.  Funded projects are focusing on a wide array of disease-relevant cell types and testing multiple technologies for delivery to these cell types.

Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-016
PI Name Institution Name Title
ASOKAN, ARAVIND DUKE UNIVERSITY Evolving High Potency AAV Vectors for Neuromuscular Genome Editing
CHEN, ZHENG-YI (contact)
LIU, DAVID R
XU, QIAOBING		 MASSACHUSETTS EYE AND EAR INFIRMARY Efficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles
DEVERMAN, BENJAMIN E BROAD INSTITUTE, INC. Novel AAVs engineered for efficient and noninvasive cross-species gene editing throughout the central nervous system
GAO, GUANG-PING (contact)
ANDERSON, DANIEL G
XUE, WEN 		 UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL WORCESTER Develop combinatorial non-viral and viral CRISPR delivery for lung diseases
GHIRAN, IONITA CALIN  BETH ISRAEL DEACONESS MEDICAL CENTER Bioengineered red blood cells as extracellular vesicle-mediated delivery platforms for gene editing machinery
GONG, SHAOQIN (contact)
EMBORG, MARINA
LEVINE, JON E
ROY, SUBHOJIT
SAHA, KRISHANU		 UNIVERSITY OF WISCONSIN-MADISON Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the Brain
MCCRAY, PAUL B UNIVERSITY OF IOWA Delivery of CRISPR Ribonucleoproteins to Airway Epithelia Using Novel Amphiphilic Peptides
SALTZMAN, W. MARK (contact)
GLAZER, PETER M 		 YALE UNIVERSITY Poly(amine-co-ester)s for Targeted Delivery In Vivo of Gene Editing Agents to Bone Marrow and Lung
SONTHEIMER, ERIK J (contact)
KHVOROVA, ANASTASIA
WATTS, JONATHAN K
WOLFE, SCOT A		 UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL WORCESTER Enhancing CRISPR Gene Editing in Somatic Tissues by Chemical Modification of Guides and Donors
Innovative Technologies to Deliver Genome Editing Machinery to Disease-relevant Cells and Tissues (UG3/UH3 Clinical Trial Not Allowed) RFA-RM-18-023
PI Name Institution Name Title
BANKIEWICZ, KRYSTOF S (contact)
MURTHY, NIREN		 UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
 Development of a nanoparticle-based gene editing technology for neurological applications
BAO, GANG (contact)
LAGOR, WILLIAM RAYMOND		 RICE UNIVERSITY Velcro AAV Vector for tissue-specific delivery of genome editing reagents with enhanced cargo capacity
CHAIKOF, ELLIOT BETH ISRAEL DEACONESS MEDICAL CENTER Delivery Technologies for In Vivo Genome Editing
CURIEL, DAVID TERRY WASHINGTON UNIVERSITY Endothelial-targeted adenovirus for organ-selective gene editing in vivo
DAHLMAN, JAMES (contact)
SANTANGELO, PHILIP J		 GEORGIA INSTITUTE OF TECHNOLOGY Highly Specific ZFN-Based HSC Gene Editing Therapies Identified By In Vivo Barcode Nanoparticle Screens And Rationally Designed Mrna
LAM, KIT S (contact)
CHENG, R.HOLLAND		 UNIVERSITY OF CALIFORNIA AT DAVIS Cell-specific nanocarrier with endocytic and endosomolytic activities for therapeutic genome editing
LEONG, KAM W COLUMBIA UNIVERSITY HEALTH SCIENCES Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders
TILTON, JOHN CHRISTIAN CASE WESTERN RESERVE UNIVERSITY In vivo delivery of CRISPR Cas9-guide RNA nucleoprotein complexes using the nanoPOD platform
WILSON, ROSS (contact)
DOUDNA, JENNIFER A		 UNIVERSITY OF CALIFORNIA BERKELEY Cas9 RNP delivery to immune cells in vivo via molecular targeting
YI, GUOHUA UNIVERSITY OF TEXAS HEALTH CENTER AT TYLER Novel CRISPR-Cas9 protein delivery to T cells in vivo by targeting CD7
ZHOU, JIANGBING YALE UNIVERSITY Novel grafted terpolymers for targeted delivery of CRISPR/Cas9- mediated precise genome editing to the brain

Animal Reporter and Testing Centers | Biological Effects Projects | Genome Editor Projects | Dissemination and Coordinating Center