Articles related to GENE THERAPY

Pediatric hearing loss care has shifted from physiologic detection and rehabilitation toward genetic diagnosis, prognostic counseling, and emerging targeted therapies. Advances in screening, cochlear implantation, and genetic testing have expanded etiologic evaluation, while therapeutic applications remain in early development.

The FDA expanded approval of the CRISPR-based gene therapy Casgevy to include patients as young as 2 years with sickle cell disease or transfusion-dependent beta thalassemia. The decision was supported by pediatric phase 3 data demonstrating sustained reductions in disease burden.

Synthetic DNA molecules known as polypurine reverse Hoogsteen hairpins reduced PCSK9 expression and lowered LDL cholesterol by approximately 50% in preclinical models, representing an early-stage gene-targeted approach distinct from existing monoclonal antibody and RNA interference strategies.

“When it is proven to be safe, durable, deliverable in young children, and clearly improves long-term communication outcomes, not just hearing thresholds, then it becomes a routine referral.” — Julie Wei, MD, Akron Children’s Hospital

“Now that we have a treatment for genetic deafness, our hope is that this will spur universal genetic testing in all kids with hearing loss.” —Lawrence Lustig, MD, CHORD study investigator

Valoctocogene roxaparvovec gene therapy demonstrates long-term efficacy in reducing bleeding episodes and factor VIII use in patients with severe hemophilia A, potentially changing the treatment landscape for this condition.