A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

A universal gene correction approach for FKRP-associated dystroglycanopathies…

Mutations within the fukutin-related protein (FKRP) gene end in a broad spectrum of muscular dystrophy (MD) phenotypes, together with the extreme Walker-Warburg syndrome (WWS). Right here, we develop a gene-editing method that replaces your complete…
Exploiting Single-Cell Tools in Gene and Cell Therapy

Exploiting Single-Cell Tools in Gene and Cell Therapy

Single-cell molecular instruments have been developed at an unimaginable tempo over the past 5 years as sequencing prices proceed to drop and quite a few molecular assays have been coupled to sequencing readouts. This fast…
Review of the Molecular Genetics of Basal Cell Carcinoma; Inherited Susceptibility, Somatic Mutations, and Targeted Therapeutics

Review of the Molecular Genetics of Basal Cell…

Basal cell carcinoma (BCC) is a major public well being concern, with greater than three million circumstances occurring every year in america, and with an growing incidence. The molecular foundation of BCC is advanced, involving…
Comprehensive Study Identifies a Sensitive, Low-Risk, Closed-System Model for Detection of Fungal Contaminants in Cell and Gene Therapy Products

Comprehensive Study Identifies a Sensitive, Low-Risk, Closed-System Model…

The US Meals and Drug Administration (FDA) regulates manufacturing and testing of superior therapeutic medicinal merchandise (ATMPs) to make sure the security of every product for human use. Gold normal sterility testing (USP<71>) and different…
Short regulatory DNA sequences to target brain endothelial cells for gene therapy

Short regulatory DNA sequences to target brain endothelial…

Gene vectors focusing on CNS endothelial cells enable to govern the blood-brain barrier and to right genetic defects within the CNS. As a result of vectors based mostly on the adeno-associated virus (AAV) have a…