gene therapy Archives - Baylor College of Medicine Blog Network

New study helps researchers engineer better gene therapies

April 1, 2025 AAV, gene therapy, improved treatments, Research, viral vectors

This study will make basic research on AAV vectors for gene delivery and preclinical gene therapy studies in mice more efficient and reproducible.

From The Labs

Not too late to repair: gene therapy improves advanced heart failure in animal model

February 13, 2024 advanced heart failure, Cardiac regeneration, gene therapy, Research

This work has important implications for the large group of patients with advanced heart failure for whom there are currently no treatments to improve their condition.

From The Labs

A novel switch to turn genes on/off on cue

January 17, 2024 gene regulation, gene therapy, polyA signal, Research, tetracycline

This new technology can effectively regulate gene expression, a promising solution to improve gene therapy clinical applications.

From The Labs

Rett patients receive a new gene therapy treatment at Texas Children’s Hospital and Baylor College of Medicine

December 12, 2023 clinical trial, gene therapy, NGN-401, Research, Rett Syndrome

Nearly 25 years after the discovery that mutations in MECP2 gene cause Rett syndrome, the first gene therapy trial for this difficult-to-treat condition is underway.

From The Labs

New research moves novel gene therapy for heart failure closer to the clinic

July 20, 2021 cardiomyocytes, gene therapy, heart attack, heart failure, heart repair, Hippo pathway, Research

Knocking down the Hippo signaling pathway in pig cardiomyocytes after a heart attack, resulted in heart tissue renewal and improved function.

From The Labs

From the labs to patent; osteoarthritis gene-therapy product is moving toward clinical trials

June 18, 2019 clinical trial, gene therapy, Il-1, Osteoarthritis, patent, Research

More than 30 million people in the United States alone are affected by osteoarthritis (OA), a degenerative joint disease, and that number is expected to

Policywise

The gene therapy watchers: Can an NIH advisory committee reinvent itself?

October 5, 2018 Center for Medical Ethics and Health Policy, Dr. Christopher Scott, FDA, gene therapy, NIH

Francis Collins, NIH Director, and Scott Gottlieb, FDA Commissioner, recently announced plans to eliminate oversight of new gene therapy experiments by the NIH’s Recombinant DNA

From The Labs

Gene therapy offers potential treatment for osteoarthritis

September 18, 2018 Flexion Therapeutics, gene therapy, GeneQuine, Il-1, Osteoarthritis, Research

Osteoarthritis is a multifactorial degenerative joint disorder that poses a major challenge for treatment. This condition currently affects 28 million people in the United States

Policywise

Gene therapy: An out-of-body experience

July 1, 2016 Affiliates, Center for Medical Ethics and Health Policy, David Vetter, Dr. Christopher Scott, gene therapy, Genetics, Healthcare, Houston, Research

On April 1, 2016, the European Medicines Agency (EMA) approved the first ex-vivo (outside the body) gene therapy in the Western Hemisphere. Diseased stem cells