Category Archives: Gene therapy

How to Sequence a Genome ?

Studying the human genome – the complete set of human genes – is a way of studying fundamental details about ourselves. The three billion letters of the human genome are written using the four-letter alphabet of DNA. The DNA is divided among 23 pairs of chromosomes that are found in each of the trillions of cells in our bodies. In 2003, The Human Genome Project produced a complete representative sequence of the human genome. Of course, people are not identical, and DNA sequences do differ subtly between individuals. Currently, a … Read more

What if mRNA could be a drug – Moderna Therapeutics

Modified messenger RNA (mRNA) Therapeutics™ can be encoded for virtually any known protein and designed to be taken up by the cells in specific tissues and organs. Our novel chemistry enables mRNA to elude the body’s innate immune response. Once delivered, like native mRNA in healthy individuals, mRNA Therapeutics™ act as cellular software directing ribosomes to express proteins or antibodies within targeted tissues – and have the power to catalyze the expression of hundreds to thousands of proteins for each mRNA molecule.

mRNA drugs drive therapeutic benefit by virtue of … Read more

How CRISPR-Cas9 Works ?


Researchers are developing ways to use CRISPR to treat genetic conditions like sickle-cell anemia and cystic fibrosis, and are also experimenting with genetic changes that could eliminate viruses like HIV. Even though viruses aren’t genetic diseases, certain gene edits have been shown to prevent the virus from spreading to new cells and to “destroy inactive HIV residing in the human genome by altering critical viral genes,” according to a look at genome surgery in MIT Tech Review. Experts even think these types of changes could eventually help treat complex conditions … Read more

Gene Therapy Technology Companies

Gene therapy is getting major attention again this month with the recently announced deal between Bristol-Myers Squibb and uniQure and upcoming top-line data from Celladon’s Phase 2 CUPID 2 study in heart failure.

We believe this renewed attention to the space could be translated into growth opportunities for companies in the field as well as a potential boost for three Buy-rated names in our universe: Applied Genetic Technologies (ticker: AGTC), GenVec ( GNVC ) and Oxford Biomedica ( OXBDF ).

Earlier in April, Buy-rated Bristol-Myers ( BMY ) and uniQure … Read more

Cystic fibrosis Cure with RNAi Technology – ProQR

What is cystic fibrosis?
Cystic fibrosis (CF) is a genetic disease affecting more than 70,000 patients worldwide. CF patients have viscous mucus accumulating in their vital organs. The mucus clogs tubes and organs thereby disrupting several processes in the body.

Pancreatic enzymes, necessary to digest food, are blocked from entering the intestines, so CF patients are not able to digest food by themselves. The thick layer of mucus in the lungs is a great environment for destructive bacteria. The thick mucus makes it hard to clear the lungs from these … Read more

Cellectis – CAR T-Cell Therapy

All you need to know about Cellectis :

4 branchs :

-Oncology
-Sceil
-Cellectis Bioresearch
-Cellectis Plants Science (CPS)

Corporate presentation : urlz.fr/1AWL


http://www.capitalpress.com/Nation_World/Nation/20141125/new-gmo-potato-avoids-usda-regulation

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http://sceil.com/
http://www.ascrnetwork.com/index.php?option=com_content&view=article&id=3014:cellectis-bioresearch-inc-wins-a-69-million-contract-with-the-national-institutes-of-health-on-ips-cells&catid=102

http://infocampo.com.ar/nota/campo/67136/cellectis-plant-sciences-una-emergente-que-puede-revolucionar-el-mejoramiento-vegetal

http://www.cellectis.com/en/content/cellectis-step-towards-new-therapy-aids-cellectis-publishes-genome-engineering-advance

http://www.nature.com/srep/2015/150130/srep08150/full/srep08150.html… Read more

CRISPR/Cas9 and Targeted Genome Editing


The development of efficient and reliable ways to make precise, targeted changes to the genome of living cells is a long-standing goal for biomedical researchers. Recently, a new tool based on a bacterial CRISPR-associated protein-9 nuclease (Cas9) from Streptococcus pyogenes has generated considerable excitement (1). This follows several attempts over the years to manipulate gene function, including homologous recombination (2) and RNA interference (RNAi) (3). RNAi, in particular, became a laboratory staple enabling inexpensive and high-throughput interrogation of gene function (4, 5), but it is hampered by providing only temporary Read more