Gene therapy corrects sickle cell disease in mice, Science authors report

This release is also in French. Please click here.

A new gene therapy method corrects sickle cell disease in mice, and may suggest future therapies to treat the genetic disease in humans. The method, developed by an international research team, is described in the 14 December issue of the journal, Science.

The therapy counteracts the faulty gene that causes red blood cells to "sickle" or deform, by transferring an anti-sickling variant of the gene to bone marrow with a viral delivery system. Once there, the anti-sickling gene incorporates itself into the stem cells that give rise to red blood cells.

In two mouse models, the new gene was rapidly expressed in 99 percent of all circulating red blood cells, preventing sickling and other signs of the disease, says study author Philippe Leboulch of the Harvard Medical School and Massachusetts Institute of Technology.

Leboulch says that there are still several obstacles to overcome before the therapy can be tested in humans, but that the method is "clearly corroborating evidence that these types of vectors based on lentiviruses are highly efficient with stable expression."

Sickle cell disorders are most common in individuals of African, Mediterranean, Indian, and Middle Eastern descent, and one in every 13 African Americans carries the sickle cell trait, according to Leboulch. The disease is caused by a single nucleotide mutation in the human beta globin gene.

Why does a mutation of the beta globin gene cause disease? Red blood cells need oxygen. In healthy people, oxygen is carried to red blood cells by hemoglobin, a molecular complex. Beta globin plays a role in getting oxygen to red blood cells, too, because it contributes two protein "chains" to hemoglobin. When a person inherits the sickle cell mutation from both parents, he or she manufactures an abnormal version of hemaglobi

Contact: Lisa Onaga
American Association for the Advancement of Science

Page: 1 2 3

Related biology news :

1. Targeted therapy knocks out pediatric brain cancer in mice
2. New anti-inflammatory strategy for cancer therapy identified by UCSD researchers
3. Columbia research to examine gene influence on severity of peridontal disease, therapy response
4. Case for IBD combination therapy comes from research at Baylor, MIT and Hebrew University
5. The search for a kinder, gentler chemotherapy
6. Embryonic stem cell therapy shows steady benefits in rebuilding infarcted heart
7. Retroviral gene therapy? ASLV, HIV, and MLV show distinct target site preferences
8. Gene changes linked to increased eye pressure may have implications for glaucoma therapy
9. Jefferson Lab detector technology aids development of cystic fibrosis therapy
10. Stroking up cellular therapy
11. Smart gene therapy protects against damage from heart attack

Post Your Comments:

(Date:6/23/2020)... ... June 23, 2020 , ... First Aid Africa, a ... UK supplier and manufacturer of innovative portable renewable energy equipment, have announced the ... against the COVID-19 pandemic in Zambia. , One of Renovagen’s FAST FOLD portable ...
(Date:6/19/2020)... ... , ... The director of stem cell biotechnology company Asymmetrex , Dr. ... pharmaceutical industries have easier access to stem cell counting, they will soon make it ... Independence Day weekend in the U.S., Asymmetrex is offering free tissue stem cell counting ...
(Date:6/11/2020)... ... June 08, 2020 , ... Cryo-Cell International’s ... measures to protect workers in the medical field. The face masks will be ... risk their lives every day to provide healthcare to pregnant women. The delivery ...
Breaking Biology News(10 mins):
(Date:7/10/2020)... ... 2020 , ... Bode Technology, a leading provider ... through its COVID-19 testing service, Bode-CARES . Bode-CARES combines high-volume clinical testing ... provides a turnkey solution that includes a comprehensive collection and virus-management program for ...
(Date:7/10/2020)... ... July 09, 2020 , ... Sentien Biotechnologies, Inc., a ... of Allen R. Nissenson, M.D., F.A.C.P., as its Chief Medical Officer. Dr. ... lead product, SBI-101. Dr. Nissenson serves as an Emeritus Professor of Medicine ...
(Date:7/1/2020)... ... June 29, 2020 , ... MedShift , a leading ... despite many obstacles created as a result of COVID-19. As a provider at ... medical practices and medical manufacturers by expanding access to device offerings, technology services, ...
(Date:6/28/2020)... ... ... The Indoor Lab, a leading provider of sensor solutions for monitoring foot ... ensure cleanliness, distancing and occupancy with its patent pending WipeAway™ technology using Artificial Intelligence ... a health performance index system that scores all aspects of compliance similar to the ...
Breaking Biology Technology:
Cached News: