Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological...

Sio Gene Therapies, Inc. (formerly known as Axovant Gene Therapies) was a clinical-stage pharmaceutical company that developed gene therapies to treat...

Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by several methods, summarized below. The two major classes of methods...

designed to address the root cause are currently available including gene therapy (Elevidys), and antisense drugs (Ataluren, Eteplirsen etc.). Other medications...

option for people with severe symptoms. There is only one FDA-approved gene therapy that is commercially available to RP patients with Leber congenital amaurosis...

cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). The gene therapy is...

This article contains a list of commercially available gene therapies. Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency...

using gene therapy as routine therapy is not suggested. A small study published in 2015 found a small benefit. The focus of much CF gene therapy research...

allele in certain populations. Researchers are looking at gene therapy or enzyme replacement therapy as possible treatments. Tay–Sachs disease is typically...

Gene therapy for color blindness is an experimental gene therapy of the human retina aiming to grant typical trichromatic color vision to individuals with...

introduction (through gene therapy) of suicide genes is a potential way of treating cancer or other proliferative diseases. Suicide genes form the basis of...

stem-cell-based therapy. Other gene therapy approaches has also been explored in the context of glioblastoma, including suicide gene therapy. Suicide gene therapy is...

desmopressin may be used in those with mild haemophilia A. Studies of gene therapy are in early human trials. Haemophilia A affects about 1 in 5,000–10...

genes: gene therapy gene therapy for epilepsy gene therapy for osteoarthritis gene therapy for color blindness gene therapy of the human retina gene therapy...

Lentiviral vectors in gene therapy is a method by which genes can be inserted, modified, or deleted in organisms using lentiviruses. Lentiviruses are...

transfusion and the medication hydroxycarbamide (hydroxyurea). In 2023, new gene therapies were approved involving the genetic modification and replacement of...

choroideremia; however, retinal gene therapy clinical trials have demonstrated a possible treatment. Since the CHM gene is located on the X chromosome...

papillomas and bladder cancer. Several drug therapies are being developed based on p53, the tumour suppressor gene that protects the cell in response to damage...

"Systemic therapy of experimental breast cancer metastases by mutant vesicular stomatitis virus in immune-competent mice". Cancer Gene Therapy. 12 (4):...

candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models. Gene therapy vectors using AAV can infect both...

"Adenovirus-Mediated Gene Delivery: Potential Applications for Gene and Cell-Based Therapies in the New Era of Personalized Medicine". Genes & Diseases. 4 (2):...

animal models of the disease and studies of the potential usefulness of gene therapy, stem cell transplants, and neuroprotective agents. To aid in earlier...

are limited. For the childhood cerebral form, stem cell transplant and gene therapy are options if the disease is detected early in the clinical course....

commonly used in human cells are germline gene therapy and the engineered nuclease system CRISPR/Cas9. Gene therapy is the delivery of a nucleic acid (usually...

untreatable. The role of CRISPR in gene therapy is to substitute exogenous DNA in place of defective genes. Gene therapy has made a huge impact and opened...

with gene therapy. Most vectors can correct the disease in the liver and spleen, and can correct brain effects with a high dosage. Gene therapy has improved...

J, Iacomini J (April 2003). "Gene therapy progress and prospects: gene therapy in organ transplantation". Gene Therapy. 10 (8): 605–611. doi:10.1038/sj...

Gene doping is the hypothetical non-therapeutic use of gene therapy by athletes in order to improve their performance in those sporting events which prohibit...

purposes, by targeting mutations to specific genes, and in gene therapy. By inserting a functional gene into an organism and targeting it to replace the...

genetic modification. This could be done in order to cure diseases (gene therapy), prevent the possibility of getting a particular disease (similarly...