Navigation Links
UCLA stem cell gene therapy for sickle cell disease advances toward clinical trials

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
University of California - Los Angeles

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:
(Date:11/12/2015)... , Nov. 11, 2015   Growing ... reliable analytical tools has been paving the way ... qualitative determination of discrete analytes in clinical, agricultural, ... are being predominantly used in medical applications, however, ... environmental sectors due to continuous emphasis on improving ...
(Date:11/9/2015)... Nov. 9, 2015  Synaptics Inc. (NASDAQ: SYNA ... announced broader entry into the automotive market with a ... the pace of consumer electronics human interface innovation. Synaptics, ... ideal for the automotive industry and will be implemented ... Europe , Japan , ...
(Date:10/29/2015)... Daon, a global leader in mobile biometric ... new version of its IdentityX Platform , IdentityX ... have already installed IdentityX v4.0 and are ... FIDO UAF certified server component as an option ... features. These customers include some of the largest and ...
Breaking Biology News(10 mins):
(Date:11/27/2015)... ... November 27, 2015 , ... Pittcon ... over 2,000 technical presentations offered in symposia, oral sessions, workshops, awards, and posters. ... a wide range of applications such as, but not limited to, biotechnology, biomedical, ...
(Date:11/25/2015)... --> ... 2020 report analyzes that automating biobanking workflow will ... long-term samples, minimizing manual errors, improving the workflow ... errors such as mislabeling or inaccurate sample barcoding ... a vital role in blood fractionation, DNA extraction, ...
(Date:11/25/2015)... and HOLLISTON, Mass. , ... Inc. (Nasdaq: HART ), a biotechnology company developing ... CEO Jim McGorry will present at the ... 1, 2015 at 2:30 p.m. PT. The presentation will ... for 30 days. Management will also be available at ...
(Date:11/25/2015)... , Nov. 25, 2015 Orexigen® Therapeutics, ... will participate in a fireside chat discussion at the ... New York . The discussion is scheduled for ... .  A replay will be available ... Contact:McDavid Stilwell  , Julie NormartVP, Corporate Communications and ...
Breaking Biology Technology: