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 said.

Kohn and colleagues found that in the laboratory the hematopoietic stem cells produced new non-sickled blood cells at a rate sufficient for significant clinical improvement for patients. The new blood cells survive longer than sickled cells, which could also improve treatment outcomes.

Sickle cell disease mostly affects people of Sub-Saharan African descent, and more than 90,000 patients in the U.S. have been diagnosed. It is caused by an inherited mutation in the beta-globin gene that makes red blood cells change from their normal shape, which is round and pliable, into a rigid, sickle-shaped cell. Normal red blood cells are able to pass easily through the tiniest blood vessels, called capillaries, carrying oxygen to organs such as the lungs, liver and kidneys. But due to their rigid structure, sickled blood cells get stuck in the capillaries.

Current treatments include transplanting patients with donor hematopoietic stem cells, which is a potential cure for sickle cell disease, but due to the serious risks of rejection, only a small number of patients have undergone this procedure and it is usually restricted to children with severe symptoms.


'/>"/>

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

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:4/11/2017)... 11, 2017 Crossmatch®, a globally-recognized leader ... today announced that it has been awarded a ... Activity (IARPA) to develop next-generation Presentation Attack Detection ... "Innovation has been a driving force within Crossmatch ... allow us to innovate and develop new technologies ...
(Date:4/5/2017)... , April 5, 2017  The Allen Institute for ... Cell Explorer: a one-of-a-kind portal and dynamic digital window ... imaging data, the first application of deep learning to ... stem cell lines and a growing suite of powerful ... for these and future publicly available resources created and ...
(Date:3/30/2017)... -- On April 6-7, 2017, Sequencing.com will host the world,s ... at Microsoft,s headquarters in Redmond, Washington ... health and wellness apps that provide a unique, personalized ... is the first hackathon for personal genomics and the ... the genomics, tech and health industries are sending teams ...
Breaking Biology News(10 mins):
(Date:6/22/2017)... PA (PRWEB) , ... June 22, 2017 , ... ... and superior results to clients throughout the biopharma and life sciences industries, continue ... the industry is seeing. Tunnell’s Kip Wolf will be speaking on “The State ...
(Date:6/22/2017)... ... June 22, 2017 , ... RURO, Inc., ... Limfinity® version 6.5, a content-packed update to the Limfinity® framework. , LimitLIS® and ... and more diverse base of customers among labs and other businesses. Limfinity® 6.5 ...
(Date:6/20/2017)... Hill, CT (PRWEB) , ... June 20, 2017 ... ... support, today announced that the CTNext board of directors has formed a Higher ... by a working group composed of institution presidents and other high-ranking representatives from ...
(Date:6/20/2017)... ... 20, 2017 , ... HorizonScan is providing food and ingredient ... likely threat to their products at the annual IFT conference in Las Vegas, ... expo attracts over 20,000 attendees representing food science professionals from over 90 countries ...
Breaking Biology Technology: