Navigation Links
UCLA stem cell gene therapy for sickle cell disease advances toward clinical trials
Date:7/1/2013

Researchers at UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have successfully established the foundation for using hematopoietic (blood-producing) stem cells from the bone marrow of patients with sickle cell disease to treat the disease. The study was led by Dr. Donald Kohn, professor of pediatrics and of microbiology, immunology and molecular genetics.

Sickle cell disease causes the body to produce red blood cells that are formed like the crescent-shaped blade of a sickle, which hinders blood flow in the blood vessels and deprives the body's organs of oxygen.

Kohn introduced an anti-sickling gene into the hematopoietic stem cells to capitalize on the self-renewing potential of stem cells and create a continual source of healthy red blood cells that do not sickle. The breakthrough gene therapy technique for sickle cell disease is scheduled to begin clinical trials by early 2014. The study was published online today ahead of press in the Journal of Clinical Investigation.

Kohn's gene therapy approach, which uses hematopoietic stem cells from a patient's own blood, is a revolutionary alternative to current sickle cell disease treatments as it creates a self-renewing normal blood cell by inserting a gene that has anti-sickling properties into hematopoietic stem cells. This approach also does not rely on the identification of a matched donor, thus avoiding the risk of rejection of donor cells. The anti-sickling hematopoietic stem cells are transplanted back into the patient's bone marrow and multiply the corrected cells that make red blood cells without sickling.

"The results demonstrate that our technique of lentiviral transduction is capable of efficient transfer and consistent expression of an effective anti-sickling beta-globin gene in human sickle cell disease bone marrow progenitor cells, which improved the physiologic parameters of the resulting red blood cells," Kohn sa
'/>"/>

Contact: Shaun Mason
smason@mednet.ucla.edu
310-206-2805
University of California - Los Angeles
Source:Eurekalert

Page: 1 2

Related biology news :

1. Neuroblastoma: Autophagy protects from chemotherapy
2. Dendritic cell therapy improves kidney transplant survival, Pitt team says
3. No danger of cancer through gene therapy virus
4. An article in Cell reveals a new resistance mechanism to chemotherapy in breast and ovarian cancer
5. New sickle cell anemia therapy advances to Phase II clinical trials
6. Posttraumatic stress disorder treatment: Genetic predictor of response to exposure therapy
7. Mount Sinai discovers new liver cell for cellular therapy to aid in liver regeneration
8. New Research: Modified Citrus Pectin - A Potent Anti-Cancer Therapy
9. Researchers reveal new more precise method of performing electroconvulsive therapy
10. Discovery may help prevent chemotherapy-induced anemia
11. Penn receives prestigious national award for breakthrough in gene therapy
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/28/2014)... FASEB MARC (Maximizing Access to Research Careers) Program ... Society of Human Genetics from October 18 22, ... meant to promote the entry of students, post doctorates ... the basic science community and to encourage the participation ... Genetics. This year MARC conferred 16 awards totaling ...
(Date:8/28/2014)... MD FASEB MARC (Maximizing Access to Research ... for the Genetics Society of America,s 27th Annual ... October 3, 2014 in Pacific Grove, California. ... of students, post doctorates and scientists from underrepresented ... community and to encourage the participation of young ...
(Date:8/28/2014)... Aug. 28, 2014  Privacy Advocate and Senior Staff ... Lynch , joins the lineup of biometric and mobile ... Biometrics UnPlugged Executive Summit in Tampa, Florida ... Previously announced speakers include Steven Rahman, Director, Technology and Strategy ... at Experian. The theme of this year,s event is ...
Breaking Biology News(10 mins):Biometrics UnPlugged Welcomes the Electronic Frontier Foundation's Jennifer Lynch to the Mobility at the Crossroads of Commerce and Privacy Summit 2
... Cells trying to keep pace with constantly changing environmental ... their genomic integrity and allowing enough genetic flexibility to ... at the Stowers Institute for Medical Research were able ... unstable, readily acquiring or losing whole chromosomes to enable ...
... today finds that tropical vegetation contains 21 percent more ... remote sensing and field data, scientists from Woods Hole Research ... able to produce the first "wall-to-wall" map (with a spatial ... of forests, shrublands, and savannas in the tropics of Africa, ...
... of rare, endemic species in the Central Andes remain ... climate change, according to a Duke University-led international study. ... particularly vulnerable to changes in the environment or climate. ... percent of the areas with high numbers of these ...
Cached Biology News:That which does not kill yeast makes it stronger 2That which does not kill yeast makes it stronger 3The good news about carbon storage in tropical vegetation 2The good news about carbon storage in tropical vegetation 380 percent of 'irreplaceable' habitats in Andes unprotected 2
(Date:8/29/2014)... Hills, NY (PRWEB) August 29, 2014 ... Burnt Hills Veterinary Hospital are seeking candidates to participate ... dogs with osteoarthritis. The ultimate goal of this ... donor stem cells into one or two arthritically affected ... treated joints. , Candidates for the current investigational ...
(Date:8/29/2014)... Boston, MA, San Antonio, TX (PRWEB) August 29, ... audited, ISO 9001:2008 and ISO 13485:2003 certified, GAMP® ... it has been awarded an international Phase II ... patients with non-Hodgkin’s lymphoma. , Throughout this trial, ... services including, but not limited to, protocol and ...
(Date:8/28/2014)... “This kit has an improved extraction efficiency and provides ... ppb,” said Mark Tess, PhD, Mycotoxin Product Manager for Charm ... and USDA-GIPSA inspection agencies that have a need to detect ... in a matter of minutes with nothing but our equipment ... and grain before accepting it, but they no longer have ...
(Date:8/28/2014)... Willow Grove, PA (PRWEB) August 28, 2014 ... talent, and create a culture that will allow individuals ... that are becoming increasingly important for employers looking to ... life science industries are embracing this trend, as the ... the fabric of a company, which will have real ...
Breaking Biology Technology:Burnt Hills Veterinary Hospital Seeks Candidates for an Investigational Study of Stem Cells for Dogs with Arthritis 2Intrinsic Imaging Awarded Phase II Clinical Trial to Assess New Treatment for Non-Hodgkin’s Lymphoma 2Klein Hersh International Leads Philadelphia Area Life Science Industry in Company Culture 2Klein Hersh International Leads Philadelphia Area Life Science Industry in Company Culture 3
... GAITHERSBURG, Md., Nov. 12 MaxCyte, Inc.,the pioneer ... it will be hosting a live discussion with ... assays for drug discovery.,The discussion is open to ... on,Thursday, November 13th from 2:00-3:00EST., One of ...
... Surgery Institute Is ... in Moscow, MOSCOW, Nov. 12 A Vermont eye ... he implanted the,Crystalens HD into a 49 year old Russian ... Randolph, Vermont performed the surgery at the,Ninth International Conference on ...
... Recommendations, BETHLEHEM, Pa., Nov. 12 ... Marketing, Infusion Systems Division and Helen,Chang, PharmD, ... Braun Medical,Inc. (B. Braun), a global leader ... B. Braun at the Institute for Safe ...
Cached Biology Technology:MaxCyte Hosts Live Discussion with Industry Experts on the Challenges in Developing Cell-Based Screening Assays for Drug Discovery 2Vermont Surgeon Implants First Accommodating Crystalens in Russia 2B. Braun Directors Participate at First ISMP National Smart Pump Safety Summit 2B. Braun Directors Participate at First ISMP National Smart Pump Safety Summit 3
... choice of PDMS Stamps (3 PDMS micropatterned ... micron squares or 100 um x 10 ... lines, 3 PDMS nano-patterned stamps with depth ... pitch of 400 nm, or 3 PDMS ...
Request Info...
RECOMBINANT EQUINE IL-4 Product Type: Recombinant Protein...
Request Info...
Biology Products: