StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

A new technology that delivers two halves of a gene separately could enable gene therapy treatments of muscular dystrophies.

Although gene therapies targeting specific genetic mutations in hematopoietic stem and progenitor cells (HSPCs) have shown ...

(Yicai) Nov. 15 -- The Germany’ Merck and five other overseas pharmaceutical companies have been given the green light to ...

The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...

In recent years, scientists have created a range of new methods based on CRISPR-Cas technology for precisely editing the ...

Join the GenScript Biotech Global Forum in London to Navigate the Future of Cell and Gene Therapies. For more information, ...

The Mass General Brigham Gene and Cell Therapy Institute, a hub of innovation dedicated to accelerating groundbreaking research, conducting clinical trials and developing FDA-approved treatments, ...

PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever ...