Compositions and methods for treatment of CEP290 related diseases are disclosed.

The present invention relates to expression constructs and viral and other vectors for the treatment and/or prevention of chronic pain.

The present invention relates to expression constructs and viral and other vectors for the treatment and/or prevention of chronic pain.

This invention pertains to mutant Lachnospiraceae bacterium ND2006 (Lb) Cas12a nucleic acids and proteins for use in CRISPR/Cas12a endonuclease systems, and their methods of use. In particular, the invention pertains to an isolated mutant LbCas12a protein, wherein the isolated mutant LbCas12a protein is active in a CRISPR/Cas12a endonuclease system. The invention also includes isolated nucleic acids encoding mutant LbCas12a proteins, ribonucleoprotein complexes and CRISPR/Cas12a endonuclease systems having mutant LbCas12a proteins.

This invention pertains to mutant Lachnospiraceae bacterium ND2006 (Lb) Cas12a nucleic acids and proteins for use in CRISPR/Cas12a endonuclease systems, and their methods of use. In particular, the invention pertains to an isolated mutant LbCas12a protein, wherein the isolated mutant LbCas12a protein is active in a CRISPR/Cas12a endonuclease system. The invention also includes isolated nucleic acids encoding mutant LbCas12a proteins, ribonucleoprotein complexes and CRISPR/Cas12a endonuclease systems having mutant LbCas12a proteins.

Liver-specific promoters and application thereof are provided. A set of small-size recombinant nucleic acid sequences for regulating high specific expression of genes in a liver system are provided. Compared with the currently reported other sequences of similar size, the said recombinant regulatory sequence fragment has the advantage that the ability of driving expression of reporter gene and human coagulation factor FVIII in the liver system is significantly enhanced, thereby being suitable for recombinant adeno-associated virus (rAAV)-mediated gene therapy.

Liver-specific promoters and application thereof are provided. A set of small-size recombinant nucleic acid sequences for regulating high specific expression of genes in a liver system are provided. Compared with the currently reported other sequences of similar size, the said recombinant regulatory sequence fragment has the advantage that the ability of driving expression of reporter gene and human coagulation factor FVIII in the liver system is significantly enhanced, thereby being suitable for recombinant adeno-associated virus (rAAV)-mediated gene therapy.

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.

Disclosed herein are compositions that comprise engineered polynucleotides, pharmaceutical compositions comprising the same, methods of making the same, and methods of treatment comprising the compositions that comprise the engineered polynucleotides.