Methods and compositions for modifying the 3′ untranslated region or coding sequence of endogenous genes using rare-cutting endonucleases and donor molecules. The methods and compositions described herein can be used to modify the coding sequence of endogenous genes or to facilitate early termination of transcripts.

The present invention relates to small interfering RNA (siRNA) molecules against the SOD1 gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS) using the siRNA molecules and AAV vectors.

Provided herein, in some aspects, are compositions of nucleic acids comprising an initial transcribed sequence and unique nucleic acid designs for high-yield and cost-effective production of ribonucleic acids.

This disclosure relates to RNA interference (RNAi) reagents for treatment of hepatitis B virus (HBV) infection, compositions comprising same, and use thereof to treat individuals infected with HBV. The reagents are artificial miRNA (shmiRNA) used alone or in combination with additional shmiRNA or shRNA.

The present invention relates generally to methods and compositions for gene therapy and immunotherapy that activate gamma delta T-cells, and in particular, can be used in the treatment of various cancers and infectious diseases.

Provided herein, in some embodiments, are materials and methods for treating hemophilia A in a subject ex vivo or in vivo. Also provided herein, in some embodiments, are materials and methods for knocking in a coding sequence encoding a synthetic FVIII having a B domain substitute into a genome.

This document relates to using bidirectional, multi-enzymatic scaffolds to biosynthesize cannabinoids in recombinant hosts.

The present invention includes compositions and methods comprising viral vector systems for multiplexed activation of endogenous genes as immunotherapy and viral-based immune-gene therapy.

This disclosure relates to the field of exosome delivery systems. In particular, compositions comprising adipose-derived exosomes that may be used as a delivery system are encompassed. The exosome delivery system can be used to deliver exogenous cargo such as miRNA and other inhibitory RNAs, as well as proteins, to target cells in a subject.

Provided is a xylanase mutant having improved specific activity, comprising any one or more mutation sites of M78F, V1431, R148K, F163W, I177V, and V206L. The specific activity of the mutant is improved, the production cost of xylanase is reduced, and the mutant can be used in feed.