Site-specific Modification of the Human Genome Using Custom-designed ZFNs

Scientists at JHU have designed sets of Zinc Finger Nucleases (ZFN) to target mouse genes, namely the tyrosinase (mTYR) and CFTR (mCFTR) and human genes, namely the CCR5 co-receptor (hCCR5) through which HN gains entry into cells early in the infection; the DMPK gene, which is involved in myotonic dystrophy; the CFTR gene, which is involved in muscular dystrophy; and �globin gene, involved in sickle cell anemia. Inverted sequences of the form (NNC/T) 3 or 4.. (G/ANN) 3or 4 separated anywhere between 4 to 6 bp appear to make excellent targets for designed ZFNs without a linker. Three finger ZFNs and four finger ZFNs were engineered to target specific sites within these genes. The efficiency of ZFN mediated gene targeting in vivo falls off rapidly with increasing spacer length greater than 6 bp. ZFNs with a linker are able to cleave such targets. The target sequence could be within a few hundred bp from the mutation site or the desired site of modification in the plant and mammalian genome for gene conversion. Description (Set) Proposed Use (Set) This invention could be utilized for targeted genome engineering of all types of cells including stern cells and whole organisms including plants and animals. This invention may be useful in human therapeutics, particularly to treat monogenic diseases (Cystic fibrosis, Sickle cell anemia and Myotonic dystrophy) in the future by gene editing or gene correction in human stern cells or somatic cells. Lastly, targeted disruption of CCRS using engineered ZFNs in hematopoietic stern cells could be used as a form of treatment for HIV in the future.

Inventor(s): Chandrasegaran, Srinivasan

Type of Offer: Licensing

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