Great Ormond Street hoping to license gene therapy for bubble baby' syndrome
Gene therapy trial had high success but was dropped by pharmaceutical company, prompting Great Ormond Street hospital to license the therapy non-profit for wider access. [ more ]
Gene therapy hailed as medical magic wand' for hereditary swelling disorder
A groundbreaking gene therapy has provided a permanent cure for patients with hereditary angioedema, a rare and debilitating disease.
Patients in the first human trial of the therapy experienced a dramatic improvement in their symptoms and were able to come off long-term medication. [ more ]
Dr. Stuart Orkin's research on identifying genes regulating fetal hemoglobin led to a revolutionary gene therapy for sickle cell disease using CRISPR technology. [ more ]
World's first gene therapy for sickle cell and thalassemia approved in the U.K.
The UK has authorized the first gene therapy treatment for sickle cell disease, offering potential relief to thousands of patients.
Casgevy, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine licensed using the gene editing tool.
Gene therapy offers a potential cure for sickle cell disease and thalassemia, which have previously only been manageable through difficult treatments. [ more ]
A Single Infusion of a Gene-Editing Treatment Lowered High Cholesterol
Researchers have shown that a single infusion of a gene-editing treatment can reduce cholesterol levels in people.
The treatment aims to permanently lower cholesterol by using Crispr to edit a gene in the liver, and initial results show significant reductions in LDL cholesterol.
Gene editing could provide a lasting option for treating hereditary high cholesterol, replacing the need for long-term medication. [ more ]
Gene therapy trials treat inherited blindness and deafness DW 05/09/2024
The article highlights the groundbreaking achievement of restoring hearing and vision through gene therapy in children with rare genetic mutations. [ more ]
Breakthrough therapies at Stanford are saving lives. Can we afford them?
Cell and gene therapies offer promising medical solutions but come with a hefty price tag, raising concerns about availability and affordability. [ more ]
Young Patient Dies After Receiving Pfizer Gene Therapy
The tragic death of a young boy during a Pfizer medical trial for Duchenne muscular dystrophy has raised concerns and questions among scientists and parents. [ more ]
Gene therapy has been successful in treating angiodema, a genetic disorder that causes painful swelling attacks.
The single-dose gene therapy using Crispr-Cas9 showed potential as a permanent cure for angiodema and has the potential to be a treatment for other genetic conditions. [ more ]
Casgevy: UK approves gene-editing drug for blood disorders
The UK has approved a gene therapy using the Crispr gene-editing tool to treat sickle cell disease and beta thalassemia, a first in the world.
The treatment involves removing stem cells from a patient's bone marrow and using Crispr to disable the faulty gene, allowing the body to produce functioning haemoglobin.
Trials have shown promising results, with the majority of patients experiencing relief from symptoms and no longer needing blood transfusions. [ more ]
In a World First, a Patient's Antibody Cells Were Just Genetically Engineered
Seattle-based biotech company, Immusoft, has genetically programmed a patient's B cells to treat a genetic disorder known as mucopolysaccharidosis type I (MPS I).
The patient's B cells are engineered to produce an essential enzyme that his body doesn't produce, eliminating the need for regular infusions of the enzyme. [ more ]
Breakthrough Gene Treatments For Sickle Cell Disease Approved
Regulators have approved two new gene therapies for sickle cell disease, offering hope for a cure for the painful blood disorder
The treatments are designed for patients 12 and older with severe forms of the disease, and one of them is the first therapy based on CRISPR gene editing [ more ]