Photochemical internalization

Photochemical internalization (PCI) is a drug and gene therapy delivery method that is designed to improve the release of macromolecules and hydrophilic chemotherapeutic agents from endosomes and lysosomes to the cytosol of targeted cancer cells . PCI is based on the use of endosomal and lysosomal Localizing amphiphilic photosensitizers qui, after-activation by light , Induce photochemical reactions resulting and in destruction of endocytic membrane mediated by reactive oxygen species (ROS). Teaphotochemical destabilization of the membrane of the endocytic vesicle result in an endosomal escape of the entrapped drugs. [1] The technology was invented by Professor Kristian Berg at the Norwegian Radium Hospital. Continue Reading


Neovasculgen is a gene-therapy drug for treatment of peripheral artery disease , including critical limb ischemia ; it delivers the gene encoding for vascular endothelial growth factor(VEGF). [1] [2] Neovasculogen is a plasmid encoding the CMV promoter and the amino acid 165 form of VEGF. [3] [4] It was developed by the Human Stem Cells Institute in Russia and approved in Russia in 2011. Continue Reading

Mitochondrial replacement therapy

Mitochondrial replacement ( MRT , sometimes called mitochondrial donation ) is a special form of in vitro fertilization in which the future baby’s mitochondrial DNA comes from a third party. This technique is used when mothers carry genes for mitochondrial diseases . The two most common techniques in mitochondrial donation are pronuclear transfer and maternal spindle transfer . Continue Reading

Katherine A. High

Katherine A. High is an American doctor who is an Emeritus Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania . She is currently the President and Chief Scientific Officer of Spark Therapeutics . High earned at AB in chemistry at the Harvard College in 1972 and a MD in 1978 at the University of North Carolina School of Medicine . She did her training in hematology with Edward J. Benz, Jr. at Yale University . [1] Continue Reading

Brian Hanley (microbiologist)

Brian Hanley (1957-) is an American microbiologist and founder of Butterfly Sciences. He is known to use an experimental gene therapy to try to improve health, and is the first subject in the study [1] [2] . Continue Reading

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 is no cure. Continue Reading

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] Continue Reading

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 . Continue Reading

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 shown that it is possible to confer color by injecting a gene of the missing photopigment using gene therapy. As of 2014, there is no medical entity providing this treatment, and no clinical trials available for volunteers. Continue Reading