New startup looking to cure genetic diseases by editing genes in new way

December 4, 2013 by Bob Yirka report

(Medical Xpress)—A new startup company called Editas Medicine (with $43 million in funding) is looking to expand on research that has already led to a system called Clustered Regularly Interspaced Short Palindromic Repeats/Cas (the Cas part is the name of a protein that is carried via RNA to the desired location in a DNA base pair) shortened to CRISPR/Cas. Such research, the team believes, will lead to a new type of gene therapy that could perhaps lead to cures for such diseases as Huntington's, sickle-cell anemia or cystic fibrosis.

Traditional gene therapy uses a technique to apply a healthy gene to a part of the DNA. That healthy gene can than override an unhealthy one and thus cure some diseases. Unfortunately, the technique doesn't work if the unhealthy gene causes disease by producing toxic proteins or if it leads to mutations that can override the healthy gene. CRISPR/Cas is better, the researchers at the new company say, because it can actually be used to replace unhealthy genes with healthy ones.

The system works by mimicking a process researchers discovered only a few years ago—some microbes they noted, use RNA to direct proteins to parts of the DNA to defend themselves. With CRISPR/Cas, the Cas9 protein is directed to a DNA base pair by RNA. Once there it sets to work editing the base pair or it can even work on larger segments. The editing process can result in repairs, an override or complete replacement of a base pair. It's that last option that sets the system ahead of the others, team members note—totally removing that cause disease and replacing them with ones that do not, could prove revolutionary to both and medicine in general.

Officials with the new startup are being coy about which diseases they will focus on first, though it appears likely they will start with those like Huntington's which are caused by a problem with a single base pair, rather than multiples.

Of course, they still have to make it all work in the way intended first. Currently, the RNA sometimes delivers the Cas9 to the wrong base pair, which is unacceptable, of course. There is also the problem of how to have the genetic edits realized throughout all of the cells in the body at the same time.

More information: www.editasmedicine.com/
www.editasmedicine.com/documen … edicine-LaunchPR.pdf

Related Stories

Recommended for you

Gene variant activity is surprisingly variable between tissues

August 21, 2017
Every gene in almost every cell of the body is present in two variants called alleles—one from the mother, the other one from the father. In most cases, both alleles are active and transcribed by the cells into RNA. However, ...

Genome analysis with near-complete privacy possible, say researchers

August 17, 2017
It is now possible to scour complete human genomes for the presence of disease-associated genes without revealing any genetic information not directly associated with the inquiry, say Stanford University researchers.

Science Says: DNA test results may not change health habits

August 17, 2017
If you learned your DNA made you more susceptible to getting a disease, wouldn't you work to stay healthy?

Genetic variants found to play key role in human immune system

August 16, 2017
It is widely recognized that people respond differently to infections. This can partially be explained by genetics, shows a new study published today in Nature Communications by an international collaboration of researchers ...

Phenotype varies for presumed pathogenic variants in KCNB1

August 16, 2017
(HealthDay)—De novo KCNB1 missense and loss-of-function variants are associated with neurodevelopmental disorders, with or without seizures, according to a study published online Aug. 14 in JAMA Neurology.

Active non-coding DNA might help pinpoint genetic risk for psychiatric disorders

August 16, 2017
Northwestern Medicine scientists have demonstrated a new method of analyzing non-coding regions of DNA in neurons, which may help to pinpoint which genetic variants are most important to the development of schizophrenia and ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.