A disgraced gene-editing scientist wants back in the lab.
China’s most infamous scientist is attempting a comeback. He Jiankui, who went to jail for three years after claiming he had created the world’s first genetically altered babies, says he remains ...
BioSpace4d
Sarepta’s Duchenne Gene Therapy Placed on Hold in Europe Pending Death Investigation
The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US ...
Gene editing reprograms colon cells to combat short bowel syndrome
Knocking out a single gene reprograms part of the large intestine to function like the nutrient-absorbing small intestine. In ...
BioPharma Dive4d
Roche halts testing of Sarepta Duchenne gene therapy in Europe
The Swiss drugmaker, which holds European rights to Elevidys, suspended three trials while researchers investigate the death ...
Express Healthcare19h
Defying the odds: How data and cell & gene therapy are rewriting the rare disease story
Javed Shaikh, Director of RWE & HEOR, Axtria, explains how harnessing next-generation sequencing and AI-powered analytics ...
Managed Healthcare Executive11d
Death of Elevidys Patient Rattles Duchenne Community
The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent ...
Gene editing with adeno-associated virus vector offers hope for hereditary deafness
An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...
EurekAlert!11d
New study reveals the curative potential of genome editing approach for genetic deafness
R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...