Groundbreaking Gene Therapy for Sickle Cell Disease Shows Promising Results
Researchers at Children’s Hospital of Philadelphia (CHOP) have published final results of a clinical trial for gene therapy targeting sickle cell disease. The study showed 96.7% of patients did not experience VOCs for at least a year, leading to FDA approval of CASGEVY in December 2023. This groundbreaking therapy, developed using CRISPR technology, has shown promising outcomes in preventing pain episodes and improving quality of life for patients.
Gene Therapy Breakthrough in Treatment of Inherited Heart Condition
Read about Tenaya Therapeutics’ breakthrough in gene therapy for ARVC, a genetic heart condition that can cause sudden death. New data published in Nature Communications Medicine shows promising results in mice, offering hope for potential future treatments for humans.
Scientists Conduct First-Ever Gene Therapy Tests in Whole Human Liver
Groundbreaking study by Children’s Medical Research Institute (CMRI) conducts first-ever gene therapy tests in whole human liver, aiming to develop more effective treatments for inherited diseases. Research addresses challenges in bringing gene therapies from lab to clinic, utilizing innovative normothermic liver perfusion system to test AAV-based therapeutics before clinical studies. Senior author expresses excitement about potential for directly assessing gene therapeutics in human liver.
Groundbreaking Study Reveals Potential for Self-Repair and Regeneration in the Human Heart
Groundbreaking study published in Nature Cardiovascular Research reveals potential for human heart self-repair and regeneration, offering hope for paradigm shift in regenerative medicine. Research challenges existing paradigm, suggesting targeting microenvironment rather than specific cell type is instrumental in healing the injured heart.
Conditional Approval Granted to Takeda’s Qdenga Dengue Vaccine
The Drug Control Authority has granted conditional approval to Takeda’s Qdenga dengue vaccine, recommended by the World Health Organization to combat the alarming spike in dengue cases. With a reported 86.3% increase in cases and 100 deaths in 2023, the approval of the vaccine is a crucial development in addressing the public health menace of dengue fever.
Gene-Editing Breakthrough Offers Hope for Sickle-Cell Disease Patients
The year 2024 marks a significant milestone in the field of biotechnology and health as the first gene-editing treatment has made its breakthrough, offering hope to patients suffering from sickle-cell disease. This revolutionary treatment, utilizing the potent DNA-snipping technology known…
RNA-Based Switch for Gene Therapy
An RNA-Based Switch for Dialing Gene Therapy Up or Down January 2, 2024 The ability to control the expression of therapeutic genes within a “therapeutic window” is crucial to ensure the development of safe and effective gene therapies, but to…