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1. Gene therapy Gene therapy is the therapeutic delivery of nucleic acid in a patient’s cells as a drug to treat disease. [1] The first attempt at human-modifying DNA Was Performed in 1980 by Martin Cline , drank the first successful nuclear gene transfer in humans, approved by the National Institutes of Health , Was Performed in May 1989. [2] The first therapeutic use of gene transfer as...
2. Gene therapy of the human retina Retinal gene therapy holds great promise in treating different forms of non-inherited and inherited blindness ....
3. Gene therapy for osteoarthritis Gene transfer strategies for medical management of osteoarthritis have attracted the attention of scientists to the complex pathology of this chronic disease. Unlike other pharmacological treatments, gene therapy targets the disease process rather than the symptoms. [1]...
4. Designer baby A designer is a human embryo that has been genetically modified , usually following guidelines set by the parent or scientist, to produce desirable traits. This is done using various methods, such as gene therapy or Preimplantation genetic diagnosis (PGD). This technology is the subject of ethical debate, bringing up the concept of genetically modified “superhumans” to replace modern humans....
5. Gene therapy for color blindness Gene therapy for color blindness is an experimental gene therapy aiming to convert congenitally colorblinds to trichromats by introducing a photopigment gene that they lack. Though partial color blindness is considered to be a disadvantage, it is a condition that affects many people, particularly males . Complete color blindness, or achromatopsia , is very rare but more severe. While never shown in humans, animal studies have...
6. Gene therapy in Parkinson’s disease Gene therapy in Parkinson’s disease is a specific neurotransmitter ( dopamine ), protect the neural system, or the modification of genes that are related to the disease. Then these cells are transplanted to a patient with the disease. There are different kinds of treatments that focus on reducing the symptoms of the disease but there...
7. Stem-cell therapy Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also used. Research is underway to develop various sources for stem cells, and to apply stem-cell treatments for neurodegenerative diseases and conditions such as diabetes , heart...
8. Gene therapy for epilepsy Gene therapy is being studied for some forms of epilepsy . [1] It relates to viral or non-viral vectors to deliver DNA or RNA to target areas where seizures arise, or to reduce the frequency of seizures . Gene therapy HAS Delivered promising results in early stage clinical trials for other neurological disorders Such As Parkinson’s disease , [2] raising the Hope that it will Become a treatment for intractable epilepsy ....
9. Strimvelis Strimvelis is the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase Deficiency), a rare disorder caused by the absence of an essential protein called adenosine deaminase (ADA ), which is required for the production of lymphocytes . Children born with ADA-SCID do not develop a healthy immune system so...
10. European Society of Gene and Cell Therapy The European Society of Gene and Cell Therapy ( ESGCT ) is a non-profit organization for educational and scientific purposes. The aim of ESGCT is to promote fundamental and clinical research in gene therapy , cell therapy , and genetic vaccines by facilitating education, the exchange of information and technology and by serving as a professional adviser to stakeholder...