The present invention provides methods and compositions comprising an adeno-associated virus (AAV) capsid protein, comprising one or more amino acids substitutions, wherein the substitutions introduce a new glycan binding site into the AAV capsid protein.

The invention provides methods to inhibit or treat brain cancers by locally inhibiting expression or activity of growth factors or growth factor receptors.

The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within an open reading frame (ORF) of the genome of at least two genotypes of the Hepatitis B virus (HBV). The present invention also encompasses methods of using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of a HBV infection, or treating hepatocellular carcinoma (HCC). Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or HCC.

The present disclosure provides compositions and methods for treating, preventing, or inhibiting diseases of the eye. In one aspect, the disclosure provides recombinant CFH FHL-1 adeno-associated virus (rAAV) vectors comprising a complement system gene.

The present disclosure relates to recombinant vectors expressing the human ND4 gene, methods of preparing recombinant vectors expressing the human ND4 gene, and uses thereof. Recombinant AAV2 vectors as disclosed herein are useful in treating Leber Hereditary Optic Neuroretinopathy (LHON), including ND4-related LHON.

This invention relates to feedback-enabled synthetic genes, polynucleotide target cassettes, vectors, and pharmaceutical compositions for the purpose of providing transgene expression in target tissues that is capable of endogenous regulation for treating disorders such as dose-sensitive intellectual ability disorders, as well as methods of making and methods of using the same.

The invention provides expression cassettes and vectors, such as viral (e.g., AAV) vectors, comprising a first nucleic acid encoding a nuclease defective Cas 9 (dCas9) polypeptide and a second nucleic acid encoding a guide polynucleotide that targets the dCas9 polypeptide to the transcriptional start site of an allele encoding a mutant huntingtin gene (HTT)-encoded protein. Also provided are pharmaceutical composition comprising the disclosed expression cassettes and vectors, as well as methods of inhibiting expression of a mutant HTT protein and of treating Huntington's Disease and symptoms associated with the disease.

A gene sequence of recombinant human type II mitochondrial dynein-like GTPase having a nucleotide sequence shown in SEQ ID NO.: 1 and uses thereof. A fusion nucleic acid comprising a nucleic acid encoding human type II mitochondrial dynein-like GTPase. A recombinant expression vector comprising the nucleic acid or a fusion nucleic acid. A transformant by which the nucleic acid or the fusion nucleic acid is introduced into a host. A non-human mammalian ADOA model based on the inactivation of the gene of type II mitochondrial dynein-like GTPase, which can effectively improve the pathological manifestations of ADOA using a recombinant expression vector encoding the human type II mitochondrial dynein-like GTPase. The expression level of the nucleic acid encoding the human type II mitochondrial dynein-like GTPase is higher, therefore, more human type II mitochondrial dynein-like GTPase can be obtained in the mitochondria, which can better treat eye diseases such as ADOA.

The disclosure relates to compositions and methods for the preparation, use, and/or formulation of adeno-associated virus capsid protein variants.

Disclosed herein is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence; a mitochondrial protein coding sequence, wherein said mitochondrial protein coding sequence encodes a polypeptide comprising a mitochondrial protein; and a 3′UTR nucleic acid sequence. Also disclosed is a pharmaceutical composition comprising the recombinant nucleic acid and a method of treating Leber's hereditary optic neuropathy (LHON) using the pharmaceutical composition.