Genome Editing with CRISPR-Cas9
Here’s a short list of some common diseases that might be curable or preventable with gene editing:
Autism
Breast cancer
Colon cancer
Hemophilia
Huntington’s disease
Marfan, Parkinson’s
Prostate cancer
Retinitis pigmentosa
Sickle cell
Skin cancer
Tay-Sachs
Wilson
Duchenne muscular dystrophy
Crohn’s
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 disease—meaning it’s 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 1—designed 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
We need to keep eye on http://elixirgen.com/ This is new company so not alto of detail but they are trying.
https://lejeuneusa.org/blog/curing-down-syndrome#.Vm8-o0orJpg
http://elixirgen.com/
http://www.nature.com/news/researchers-turn-off-down-s-syndrome-genes-1.13406
I will keep this in my alert list.
http://hereandnow.wbur.org/2013/07/17/therapy-down-syndrome… Read more
For the second episode of “Science Happens,” we hung out with Pamela Silver, a scientist who wants to hack the microbiome.The microbiome — those trillions of bacteria that occupy our bodies — has become an obsession for many scientists in recent years. They are using powerful DNA-sequencing technologies to catalog the thousands of species that live in us and on us. Those experiments are revealing just how much we depend on the microbiome for our well-being. Our inner ecosystem turns out to help us digest food, influence our metabolism, nurture … Read more
Smart Jewelry … Read more
