Gene Therapy

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 collection of optimized therapeutic prime-editing strategies targeting four common splicing mutations in the peripherin‐2 gene (PRPH2) that are associated with various retinal degenerations.

This technology is an RNA templated genome editing technique that can be utilized in any organism, without the typical requirement for homologous recombination.

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 applications in other neurogenerative diseases.

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 method of reprogramming cellular metabolism to treat neurodegenerative disorders by downregulating catabolism and upregulating anabolism in cells.

This technology utilizes anti-CRISPR proteins for efficient spatial and temporal control of gene editing.

A spinal motor neuron-specific enhancer for cell-type specific gene expression control in the developing and adult central nervous system.

This technology utilizes exosomes or extracellular vesicles to deliver the CRISPR/Cas9 gene editing system for the treatment of human diseases.

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 is an AAV-mediated BEST1 gene therapy for the treatment of retinal degenerative disorders associated with Bestrophin-1 (BEST1) dominant mutations.

This technology uses CRISPR-Cas gene editing to disrupt and correct ocular disease-associated mutated genes, resulting in improved protein function and disease treatment.

This technology is a naturally occurring non-coding RNA that regulates the SLC2A1 gene, allowing for therapeutic regulation of Glut1 for treatment of diseases in which Glut1 is under-expressed.

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 DNA editing platform with a reduced payload size to allow adeno-associated virus (AAV) and nanoparticle packaging for gene therapy.