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Experiment: Validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing.

PI:   Jason D Heaney, PhD 
 

Quantification of CRISPR/Cas editing in liver and heart following custom AAV-mediated delivery. Detection of editing in non-target tissues.
Editing Assay: - Fluorescent imaging for tdTomato+ cells
- Editing efficiency calculated by counting fluorescent cells (RFP) compared to DAPI stained nuclei

Organ System Overview


 
Saline Neg control, Male
Saline Neg control, Female
cc47-SaCas9, Male
cc47-SaCas9, Female
cc47-Cre Pos control, Male
cc47-Cre Pos control, Female
Legend:  
Delivery Efficiency
Editing Efficiency
Target Tissue
Not Available
 

 
Analyze Data Sets Available for this Experiment
Connective Tissue
  Brown adipose tissue (unspecified)
  Gonadal fat pad (unspecified)
  Subcutaneous adipose tissue (unspecified)
Reproductive
  Uterus (unspecified)
  Epididymis (unspecified)
  Testis (unspecified)
  Ovary (unspecified)
Renal/Urinary
  Kidney (unspecified)
Endocrine
  Pancreas (unspecified)
Haemolymphoid
  Spleen (unspecified)
Gastrointestinal
  Duodenum (unspecified)
  Colon (unspecified)
  Ileum (unspecified)
  Jejunum (unspecified)
  Stomach (unspecified)
Liver and Biliary
  Liver (hepatocyte)  (TARGET)
Respiratory
  Lung (unspecified)
Cardiovascular
  Heart (cardiac muscle cell)  (TARGET)
Musculoskeletal
  Diaphragm (unspecified)
  Extensor digitorum longus (unspecified)
  Gastrocnemius (unspecified)
  Soleus muscle (unspecified)
  Transversus abdominis muscle (unspecified)
Nervous
  Brain (unspecified)
Sensory
  Eye (unspecified)