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gene therapy

Featured Researcher – Nizar Y. Saad, PhD
 Featured Researcher – Nizar Y. Saad, PhD 150 150 Gina Vitale
By: Gina Vitale

Not all genetic diseases are inherited; however, they are all the result of DNA mutations or flaws. Genetic research and gene therapy are integral in finding cures or helpful treatments for diseases such as muscular dystrophy. At the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital, research scientists are engaged in precedent-setting transitional gene…

Challenges in Clinical Decision-Making for Patients with Spinal Muscular Atrophy
 Challenges in Clinical Decision-Making for Patients with Spinal Muscular Atrophy 1024 683 Mary Bates, PhD
By: Mary Bates, PhD

There are three approved genetic therapies for SMA, but little guidance in treatment selection. Spinal muscular atrophy (SMA) was once a leading cause of inherited infant death in the United States. Today, there are three genetic therapies approved by the Food and Drug Administration to treat SMA — but no clinical trials directly comparing treatment…

New Study Demonstrates Potential for a Gene Therapy for Vanishing White Matter Disease
 New Study Demonstrates Potential for a Gene Therapy for Vanishing White Matter Disease 1024 552 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

The research, conducted in mouse models, shows promising safety and efficacy for a currently incurable, life-limiting condition. Researchers at Nationwide Children’s Hospital have confirmed initial safety and efficacy data for a gene supplementation therapy for Vanishing White Matter (VWM) disease in mouse models. Testing multiple versions of therapy in two separate mouse models representing different…

At the Heart of a First R01 Grant
 At the Heart of a First R01 Grant 150 150 Alaina Doklovic
By: Alaina Doklovic

Allison Bradbury, MS, PhD, is a principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital and an assistant professor in the Department of Pediatrics at The Ohio State University. She recently received her first R01 grant to develop novel therapeutic strategies for tubulin folding cofactor D (TBCD)-related developmental and epileptic encephalopathy. This…

Clinical Trial Shows Promising Results for AAV Gene Therapy for LGMD 2E/R4
 Clinical Trial Shows Promising Results for AAV Gene Therapy for LGMD 2E/R4 1024 783 Abbie Miller
By: Abbie Miller

Recently published in Nature Medicine, interim results from a Phase 1/2 trial offer promising results.   Limb-girdle muscular dystrophy (LGMD) 2E/R4 is a rare, progressive neuromuscular disorder caused by mutations in the β-sarcoglycan (SGCB) gene. These mutations lead to SGCB protein deficiency, ultimately resulting in muscle loss. Progressive, debilitating weakness and wasting begin in the…

Gene Therapy for the Masses?
 Gene Therapy for the Masses? 1024 683 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Long-lived financial and logistical hurdles make bringing new gene therapy products to market a major challenge. To help bring more of these medical miracles to fruition, experts across industry, regulatory review, science and medicine have begun to problem solve together.  With the approval of the gene therapy Kymriah® (tisagenlecleucel) in 2017, the Food and Drug…

Innovative Gene Therapy Approach for Treating Patients with Tuberous Sclerosis Type 2
 Innovative Gene Therapy Approach for Treating Patients with Tuberous Sclerosis Type 2 150 150 Lauren Dembeck
By: Lauren Dembeck

Tuberous sclerosis complex (TSC) is a devastating genetic disease that affects nearly 1 in every 5,500 newborns and approximately 2 million people worldwide. The disease is characterized by the formation of non-malignant tumors throughout multiple organs, including the kidney, lungs, eyes, and heart, but predominantly the brain. It is typically diagnosed in infants and young…

FDA Approves Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
 FDA Approves Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy 1024 632 Abbie Miller
By: Abbie Miller

On June 22, 2023, the FDA announced accelerated approval for SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in the Center for Gene Therapy at Nationwide Children’s Hospital. The FDA approved the treatment for pediatric patients 4-5 years old with DMD. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at…

Going Viral: The AAV Approach to Curing Cancer
 Going Viral: The AAV Approach to Curing Cancer 1024 256 Emily Siebenmorgen
By: Emily Siebenmorgen

According to Timothy Cripe, MD, PhD, chief of the Division of Hematology and Oncology at Nationwide Children’s Hospital, it’s an incredible time to be working on cancer treatment – and now, targeted cancer prevention. “There’s so much going on in the cancer world these days,” says Dr. Cripe, who is also a principal investigator in…

Micro-dystrophin: A Small Gene With Big Promise
 Micro-dystrophin: A Small Gene With Big Promise 1024 632 Abbie Miller
By: Abbie Miller

SRP-9001 for Duchenne muscular dystrophy supplies a functional dystrophin gene via AAVrh74 gene therapy. In 1969, Jerry Mendell, MD, was working at the National Institute of Neurological Disorders and Stroke (NINDS) when he saw his first patient with Duchenne muscular dystrophy (DMD). DMD, a severe form of muscular dystrophy caused by a mutation in the…

A Single-Dose Cancer Immunotherapy via Gene Transfer
 A Single-Dose Cancer Immunotherapy via Gene Transfer 150 150 Mary Bates, PhD
By: Mary Bates, PhD

Researchers at Nationwide Children’s Hospital report proof-of-principle results for a new gene therapy cancer treatment. The off-the-shelf, single-dose immunotherapy serves as an alternative to CAR-T therapy and can be engineered to be on-demand. T cells redirected to cancer cells either via a chimeric antigen receptor (CAR-T) or a bispecific molecule have been game-changers in treating…