RNAi.technology – RNAi Medicine, RNAi Developments, Gene …

Optogenetics From Wikipedia, the free encyclopedia Optogenetics (from Greek optiks], meaning seen, visible) is a biological technique which involves the use of light to control cells in living tissue, typically neurons, that have been genetically modified to express light-sensitive ion channels. It is a neuromodulation method employed in neuroscience that uses a combination of techniques from optics and genetics to control and monitor the activities of individual neurons in living tissueeven within freely-moving animalsand to precisely measure the effects of those manipulations in real-time.[1] The key reagents used in optogenetics Read more

GeneQuine develops drugs that are based on gene therapy. The concept of gene therapy is to introduce genetic material into the patients own cells in the body. The cells produce then a therapeutic protein according to the template that the introduced genetic material (DNA) provides

GeneQuine is focused on the development of gene therapy agents for the treatment of osteoarthritis. Osteoarthritis is a degenerative joint disorder characterized by cartilage loss and inflammation. Patients affected by osteoarthritis experience joint pain as well as swelling and stiffness of the joints leading to Read more

Genome Editing with CRISPR-Cas9

Heres a short list of some common diseases that might be curable or preventable with gene editing:

Autism Breast cancer Colon cancer Hemophilia Huntingtons disease Marfan, Parkinsons Prostate cancer Retinitis pigmentosa Sickle cell Skin cancer Tay-Sachs Wilson Duchenne muscular dystrophy Crohns Color blindness Cystic fibrosis Down syndrome Polycystic kidney Turner syndrome.

There are hundreds of other more rare genetic disorders. Read more

Gene therapy is a well-suited approach for the treatment of SMA due to the monogenic nature of the diseasemeaning its caused by the deletion of or mutations in a single gene. AVXS-101 is our clinical-stage, proprietary gene therapy candidate of a one-time, intravenous treatment for SMA Type 1designed to prevent further muscle degeneration caused by SMA through:

Delivery of a fully functional human SMN gene into target motor neuron cells Production of sufficient levels of SMN protein required to improve motor neuron function Rapid onset of effect in addition to Read more

Advantagene Inc. Bluebird Bio Genethon Human Stem Cells Institute Oxford BioMedica Plc Sanofi Shanghai Sunway Biotech Co. Ltd. Sibiono GeneTech Co. Ltd. Spark Therapeutics, LLC UniQure N.V. Vical Inc. ViroMed Co. Ltd. dba VM BioPharma Read more

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RNAi.technology - RNAi Medicine, RNAi Developments, Gene ...