This technology is a gene-targeting therapeutic approach that suppresses Cdkn2a expression to prevent premature muscle stem cell aging for spinal muscular atrophy (SMA) and other myopathies.
This technology is a collection of optimized therapeutic prime-editing strategies targeting four common splicing mutations in the peripherin‐2 gene (PRPH2) that are associated with various ...
This technology is a gene therapy platform that selectively re-expresses the developmental transcription factors Islet1 and Lhx3 in adult motor neurons, restoring youthful resilience and ...
This technology is a modified CRISPR/Cas9 gene editing therapy that is mutation-independent for treating autosomal dominant disease-related genes, including retinitis pigmentosa.
This technology is a nucleic acid-based polymer system that can be engineered to target the expression of specific cancer-associated genes for therapeutic benefit.
This technology is a variant of the synaptic chaperon protein Hspa8, which potentiates neurotransmission to suppress spinal muscular atrophy (SMA) phenotypes in SMA model mice, with further ...
This technology is an AAV-mediated BEST1 gene therapy for the treatment of retinal degenerative disorders associated with Bestrophin-1 (BEST1) dominant mutations.
This technology is a computational algorithm for code prediction and target site matching of RNA-binding pentetricopeptide repeat (PPR) proteins called PPRDecoder.
This technology is a gene therapy which delivers CRB1 isoforms to their cell-specific localizations, for the treatment of retinopathies caused by CRB1 mutations.
This technology describes compositions which enhance the potency and stability of nucleic acid therapeutics for the treatment of diseases or conditions.
This technology is a generally applicable gene therapy approach that restores metabolic dysregulation and treats retinal degenerative diseases regardless of genetic variation.
This technology is a method of reprogramming cellular metabolism to treat neurodegenerative disorders by downregulating catabolism and upregulating anabolism in cells.
This technology is a gene therapy strategy to treat TK2 deficiency-associated mitochondrial DNA depletion syndromes with an engineered adenovirus-associated viral (AAV) vector to deliver a ...
