Gene editing target disease ?


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 for spinal muscular atrophy (SMA) Type 1

Spinal-muscular-atrophy

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

New Genetic Therapy Could Erase Down Syndrome

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

Peek inside a lab trying to create living medicine

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