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Gene therapy may be classified into two types by the type of cell it affects: somatic cell and germline gene therapy. In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete, germ cell, gametocyte, or undifferentiated stem cell.
Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [13] The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile ...
With the discovery of various types of immune-related disorders, there is a need for diversification in prevention and treatment. Developments in the field of gene therapy are being studied to be included in the scope of this treatment, but of course more research is needed to increase the positive results and minimize the negative effects of gene therapy applications. [27]
One of the major challenges facing gene therapy — a way to treat disease by replacing a patient’s defective genes with healthy ones — is that it is difficult to safely deliver therapeutic ...
Medical genetics is the branch of medicine that involves the diagnosis and management of hereditary disorders.Medical genetics differs from human genetics in that human genetics is a field of scientific research that may or may not apply to medicine, while medical genetics refers to the application of genetics to medical care.
Notably, some health experts have said that high costs and logistical issues, among other factors, have limited the uptake of Hemgenix and another approved gene therapy for the more common ...
The therapy known as Casgevy [9] works through editing a dysfunctional protein that interferes with creation of adult hemoglobin. This gene is known as the BCL11A, and when people have Beta thalassemia, their bodies do not make enough adult hemoglobin. Casgevy uses precise gene editing of stem cells, and reduces the activity of BCL11A.
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]