Abstract: The disclosure described herein provides recombinant adeno-associated virus (rAAV) viral particle comprising an AAV9 serotype capsid and a vector genome encoding an RPE65 (e.g., hRPE65) polypeptide, as well as related compositions and uses thereof (e.g., use to treat Leber's congenital amaurosis 2 (LCA2)).

Abstract: Provided are an AAV capsid protein mutant and an encoding nucleic acid, and a corresponding vector and a host cell thereof. Also provided are an adeno-associated virus vector containing the AAV capsid protein mutant, a recombinant adeno-associated virus particle constructed therefrom and carrying a gene expression cassette, a preparation method therefor, and the use thereof in treating diseases.

Abstract: The invention provides novel engineered CRISPR/Cas effector enzymes, such as Cas13 (e.g., Cas13e) that substantially maintain guide-sequence-specific endonuclease activity and substantially lack guide-sequence-independent collateral endonuclease activity compared to the corresponding wild-type Cas. Also provided are polynucleotides encoding the same, vectors or host cells comprising the polynucleotides or engineered Cas, and method of use, such as in RNA-based target gene transcript knock down.

Abstract: The invention provides novel engineered CRISPR/Cas effector enzymes, such as Cas13 (e.g., Cas13d, Cas13e, or Cas13f) that substantially maintain guide-sequence-specific endonuclease activity and substantially lack guide-sequence-independent collateral endonuclease activity compared to the corresponding wild-type Cas. Also provided are polynucleotides encoding the same, vectors or host cells comprising the polynucleotides or engineered Cas, and method of use, such as in RNA-based target gene transcript knock down.