FIRST SUCCESSFUL GENE THERAPY REPORTED FOR INHERITED DISEASEGina Kolata The New York Times
It is worth celebrating that this is the first gene editing treatment for thalassemia in Asia and the first successful treatment of β0/β0 thalassemia major by CRISPR gene editing technology in the world. The patient's family and the principal investigator of the clinical study, Professor Bin...
Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology. Patients' hematopoie...
Following successful engraftment of modified HSCs in the bone marrow, these cells start to produce daughter blood cells which contain the IDS gene and enzyme which are distributed throughout the body, including the brain. Professor Bigger said, "This is a next generation stem cell gene therapy ap...
(Medical Xpress) -- The first patient to receive gene therapy for an incurable type of blindness was treated at the John Radcliffe Hospital in Oxford this week as part of a trial led by Oxford University. If successful, the advance could lead to the first-evertreatmentfor choroideraemia, a...
However, for successful treatment of LS, further improvement is needed to express mGHR to extra-hepatic tissues using ubiquitous promoters/enhancers such as CAG (CMV enhancer/chicken beta Actin promoter/rabbit beta-Globin intron) promoter to target its expression to more tissues, especially to ...
Also speaking at the press conference, Dr Aiuti explained that previous attempts at gene therapy for ADA-SCID have not been totally successful, as the children continued to need enzyme-replacement therapy (with pegylated ADA). One of the innovations in their own protocol was the use of the cy...
Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ: Successful reconstitution of immunity in ADA- SCID by stem cell gene therapy following cessation of PEG- ADA and use of mild preconditioning...
SUZHOU,China,March 21, 2024/PRNewswire/ -- NeuExcell Therapeutics, a leading biotechnology company focused onin vivoneural regenerative therapies, announces the successful dosing of the first patient with the first-in-class NeuroD1 gene therapy product NXL-004. ...
Yet, despite the knowledge we have accumulated, we haven’ t been so successful in distributing it to where it’s needed most. According to the World Bank, twelve percent of the world’s population lives on less than $ 2 a day. Malnutrition kills more than 3 million children annually. ...