The present application relates to nanoparticles for the targeted delivery of CRISPR/Cas13 systems, and their therapeutic use to treat diseases and disorders such as prostate cancer and COVID-19.

The present disclosure is broadly concerned with the field of cancer immunotherapy. For example, the present invention generally relates to an immune cell comprising a genetically engineered antigen receptor that specifically binds to a target antigen and a genetic disruption agent that reduces or is capable of reducing the expression in the immune cell of two genes that weaken the function of the immune cell.

The present disclosure relates to targeting of miRNA to activate expression of genes on the inactivated X chromosome. This gene therapy is useful for treating X-linked disorders, including Rett syndrome.

Aspects of the disclosure relate to compositions and methods for regulation of transgene (e.g., miRNAs, shRNAs or coding sequences) expression from viral vectors. In some embodiments, the disclosure provides expression constructs comprising a viral vector encoding one or more transgenes, the expression of which is regulated by a rapamycin/rapalog-based system.

An object of the present invention is to establish a cell line that is useful as a host cell for use in recombinant protein production, highly expresses transgenes stably, and grows stably. The present invention provides a method for establishing a cell line for recombinant protein production capable of stably expressing two or more foreign genes, comprising transferring a gene of a non-coding RNA suppressing the expression of NfkBia to a cell.

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.

The present invention relates to adeno-associated viral (AAV) particles modulatory polynucleotides encoding at least one siRNA molecules and methods of use thereof.

The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS).

RNA interference-based methods and products for inhibiting the expression of pathogenic dynamin-1 variants are provided. Delivery vehicles such as recombinant adeno-associated viruses deliver DNAs encoding RNAs that inhibit the expression of the dynamin-1 variants. The methods treat, for example, developmental and epileptic encephalopathies.

The present disclosure provides plant transformation vectors, T-DNA insert regions, and transformed plants. The vectors are designed to be a binary vector for use in plant transformations for such as potato. The transformed plants are characterized in that they contain the T-DNA insert region comprising stacked expression cassettes and the corresponding phenotype. The present disclosure also provides methods for identifying genetic material in transformed plants, including in food products made from such plants. The disclosure further relates to the materials and/or means for detecting plant transformation events and methods for detecting presence of plant transformation events.