Navigation Links
Genetically corrected blood cells obtained from skin cells from Fanconi anemia patients

A collaboration research carried out by the teams of Jordi Surralls, Universitat Autnoma de Barcelona (UAB); Juan Carlos Izpisa-Belmonte and ngel Raya, Centre for Regenerative Medicine of Barcelona (CMRB); and Juan Antonio Bueren, Centre for Energetic, Environmental and Technological Research (CIEMAT), has resulted in the generation of blood cells from skin cells of patients with a genetic disease known as Fanconi anemia. The process is based on gene therapy and cell reprogramming techniques in which cells similar to embryonic stem cells known as induced pluripotent stem (iPS) cells can be generated. The research article was published in this week's digital version of Nature.

The research demonstrates that, for the first time, in the case of a genetic disease such as Fanconi anemia it is possible to correct the genetic defect in patient-specific skin cells by converting them into cells similar to embryonic stem cells (iPS cells) which later can be differentiated towards blood cells.

These results are the proof of concept that this new therapeutic strategy has the potential of generating tissues using the very skin of those affected with these genetic diseases. This observation is particularly important in diseases such as Fanconi anemia, where one of the main problems lies in the lack of blood cell in the bone marrow of those affected. However, according to researchers, this new therapeutic strategy can be applied to many other genetic diseases by differentiating iPS cells towards healthy tissues these patients lack.

The generation of blood cells in this research was carried out in vitro, in cell culture plates, which places the research in a preclinical environment. It remains unknown whether they would generate blood cells after being transplanted. Moreover, the transplant of embryonic stem cells in animals has revealed that these cells can cause tumours. Therefore, the possibility of treating Fanconi anemia patients by transplanting iPS cells must wait until the efficacy and safety of these new discoveries are demonstrated in experimental models.

Researchers taking part in the study are confident that in the next few years it will be possible to improve the efficacy and safety of this new scientific discovery, and that some time in the future, clinical professionals will be able to cure patients suffering from genetic diseases such as Fanconi anemia.


Contact: Maria Jesus Delgado
Universitat Autonoma de Barcelona

Related biology news :

1. Penn Veterinary Medicine report new strategy to create genetically-modified animals
2. Study shows genetically engineered corn could affect aquatic ecosystems
3. Genetically engineered corn may harm stream ecosystems
4. Genetically modified carrots provide more calcium
5. Is political orientation transmitted genetically?
6. Total, genetically-based recall
7. Novel bioreactor enhances interleukin-12 production in genetically modified tobacco plants
8. Researchers genetically link Lou Gehrigs disease in humans to dog disease
9. Scientists unlock possible aging secret in genetically altered fruit fly
10. Cats eye diseases genetically linked to diseases in humans
11. Origins of wolverine in California genetically verified
Post Your Comments:
(Date:4/26/2016)... Research and Markets has announced ... 2016-2020"  report to their offering.  , ,     (Logo: ... analysts forecast the global multimodal biometrics market to ... period 2016-2020.  Multimodal biometrics is being ... the healthcare, BFSI, transportation, automotive, and government for ...
(Date:4/15/2016)... 15, 2016  A new partnership announced today ... underwriting decisions in a fraction of the time ... and high-value life insurance policies to consumers without ... With Force Diagnostics, rapid testing (A1C, Cotinine and ... (blood pressure, weight, pulse, BMI, and activity data) ...
(Date:4/14/2016)... April 14, 2016 BioCatch ... Detection, today announced the appointment of Eyal Goldwerger ... role. Goldwerger,s leadership appointment comes at a ... of the deployment of its platform at several of ... technology, which discerns unique cognitive and physiological factors, is ...
Breaking Biology News(10 mins):
(Date:6/23/2016)... 2016 /PRNewswire/ - FACIT has announced the creation ... biotechnology company, Propellon Therapeutics Inc. ("Propellon" or "the ... a portfolio of first-in-class WDR5 inhibitors for the ... WDR5 represent an exciting class of therapies, possessing ... for cancer patients. Substantial advances have been achieved ...
(Date:6/23/2016)... YORK , June, 23, 2016  The Biodesign ... to envision new ways to harness living systems and ... Modern Art (MoMA) in New York City ... than 130 participating students, showcased projects at MoMA,s Celeste ... Paola Antonelli , MoMA,s senior curator of architecture ...
(Date:6/23/2016)... , June 23, 2016 Apellis ... Phase 1 clinical trials of its complement C3 ... single and multiple ascending dose studies designed to ... (PD) of subcutaneous injection in healthy adult volunteers. ... (SC) either as a single dose (ranging from ...
(Date:6/23/2016)... ... June 23, 2016 , ... ... regulatory and technical consulting, provides a free webinar on Performing Quality ... 13, 2016 at 12pm CT at no charge. , Incomplete investigations are still ...
Breaking Biology Technology: