The Medical University of South Carolina Health has established the Rena N. Grant Sickle Cell Center to help treat those ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

2024 — Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and perform its gene editing ... Single-Dose Gene Therapy Is ...

Recent research takes aim at the a variant in gene SCN1B, which causes a severe form of developmental epileptic encephalopathy.

Older women could be vulnerable to harmful inflammation from new gene therapies to treat incurable eye diseases, new research has found.

Real-world data highlight gaps in genetic testing and targeted therapy use for metastatic castration-resistant prostate ...

MaxCyte, Inc., a leading, cell-engineering focused company providing solutions to advance the discovery, development and commercialization of next-generation cell therapeutics announced today the ...

The public-private partnership aims to create a sustainable, skilled talent pipeline to support the state’s rapidly expanding ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

Although these diseases were initially treated by transplantation of allogeneic hematopoietic stem cells, the past 20 years has shown that these conditions are correctable by gene therapy.

With the addition of Casgevy, Children’s Hospital Los Angeles now offers two gene therapies for sickle cell, the first being Lyfgenia.