Gene Therapy: Revolutionizing Medicine By Tackling Diseases At Their Genetic Roots
Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...
What a $2 Million Per Dose Gene Therapy Reveals About Drug Pricing
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Gene therapy for rare epilepsy shows promise in mice
Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...
News Medical on MSN3d
One shot to lower cholesterol for life? Scientists unlock a groundbreaking gene therapy
Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...
MedPage Today7d
Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
News Medical on MSN7h
Gene therapy shows promise in treating SCN1B-linked epileptic encephalopathies
Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...
Medscape8d
Could Gene Therapy for RA Soon Be Ready to ‘Take Off’?
Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...
Precision gene therapy for liver disorders increases number of correctly repaired cells
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...
Nanomachines loaded with wine ingredients overcome gene therapy challenges
Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...
GlobalData on MSN2d
GenSight’s Optic Neuropathy gene therapy sustains efficacy over five years
The five-year trial saw patients who received a single injection of Lumevoq sustaining clinical recovery in a rare condition ...
The American Journal of Managed Care21h
Mitigating Barriers to Cell and Gene Therapy Access
Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, ...
GEN2d
ExpressionEdits Partners with Boehringer to Enhance Gene Therapies with AI-Based Intron Design
Boehringer will leverage ExpressionEdits' Genetic Syntax Engine which generates optimized introns to increase protein expression.