Viral vectors, lentiviral particles, and modified cells are disclosed. They encode or express a small RNA capable of targeting the KIF11 gene. In embodiments, the viral vectors and lenti viral particles further comprise and a KIF11 gene whose non-coding region has been modified such that it is resistant to activity by the small RNA.

The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.

The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.

Compositions, recombinant viruses, and recombinant viral vectors for inhibiting sarcolipin (SLN) expression or activity in a cell and for preventing or treating Duchenne Muscular Dystrophy (DMD) in a subject (e.g., a human patient having or predisposed to having DMD) and in some embodiments additionally cardiomyopathy, include a therapeutically effective amount of an inhibitor of SLN. Methods of using these compositions, recombinant viruses, and recombinant viral vectors are also described herein. These compositions, recombinant viruses, and recombinant viral vectors and methods of use provide novel therapies for DMD and associated cardiomyopathy based on the reduction of SLN expression and/or activity.

This invention relates to compositions and methods for treating a disease or disorder associated with premature termination codon. Certain aspects of the invention relate to polynucleotides, vectors, and host cells, and uses thereof.

Provided herein are recombinant negative-strand RNA virus genomes (e.g., recombinant rabies virus genomes) and recombinant negative-strand RNA viruses (e.g., recombinant rabies viruses) and methods for their use in delivering a guide RNA and, optionally, a transgene, into a target cell. Also provided are packaging systems and methods of using the packaging systems to produce recombinant negative-strand RNA viruses.

Modified viral genomes are able to reduce induction of inflammatory and immune anti-viral responses. This manifests itself in reduced NF-kB activity, increased viral transduction rates, and increased expression of transgenes. Viral genomes are modified by incorporating one or more oligonucleotide sequences which are able to bind to TLR9 but not induce activation of it. The oligonucleotide sequences may be synthetic, bacterial, human, or from any other source.

The invention relates to a minimal U6 pol III promoter. The invention also concerns a nucleic acid construct comprising the minimal U6 pol III promoter, a vector comprising the minimal U6 pol III promoter, methods involving the minimal U6 pol III promoter, and uses for the minimal U6 pol III promoter.

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

This document relates to methods and materials for treating cancer. For example, engineered viruses (e.g., oncolytic viruses) encoding one or more inhibitors of apolipoprotein B editing complex 3B (APOBEC3B) polypeptide activity or expression and methods for using such viruses as an oncolytic agent (e.g., to treat cancer) are provided. For example, one or more engineered oncolytic viruses encoding one or more inhibitors of APOBEC3B polypeptide activity or expression can be administered to a mammal having cancer to treat that mammal.