Navigation Links
Potential gene therapy approach to sickle cell disease highlighted at American Society of Hematology
Date:12/11/2012

Boston, Mass.Researchers at Dana-Farber/Children's Hospital Cancer Center (DF/CHCC) have taken the first preliminary steps toward developing a form of gene therapy for sickle cell disease. In an abstract presented on Dec. 10 at the 54th annual meeting of the American Society of Hematology, the research teamled by DF/CHCC's Raffaele Renella, MD, PhD, Stuart H. Orkin, MD, and David A. Williams, MDannounced that they had demonstrated in an animal model the feasibility of activating a form of hemoglobin unaffected by the sickle cell mutation.

The study was included as part of the meeting's Best of ASH session on Dec. 11. At this session, the meeting's scientific co-chairs present what they consider to be the best abstracts of the 4,000-plus presented at the conference.

"This work builds on the transformative basic research discovery of the role of BCL11A in maintaining fetal hemoglobin silencing by the Orkin laboratory with a near-term goal of curing sickle cell disease using gene therapy," said Williams, chief of the Division of Hematology/Oncology at DF/CHCC and senior author on the abstract. "We have had important recent successes in applying this type of gene therapy in treating several other genetic diseases at Boston Children's Hospital."

The team's work builds on previous research, published by Orkin's laboratory, suggesting that strategies targeting a molecular switch called BCL11A have the potential to correct sickle cell disease.

First described over 100 years ago, sickle cell disease (or sickle cell anemia) is an inherited blood disease caused by a single mutation in one of the components of hemoglobin, the oxygen-carrying protein in red blood cells. The mutation reduces the protein's ability to carry oxygen and forces the cells to curve into a distinctive crescent or sickle shape.

Our bodies can actually manufacture two forms of hemoglobin: the adult form susceptible to the sickle cell mutation, and a fetal form that is largely produced during development and for a short time after birth. BCL11A is a transcription factor that facilitates that shift in hemoglobin production. Fetal hemoglobin expression significantly reduces the symptoms and complications of sickle cell disease because it prevents the intracellular abnormalities caused by the sickle mutation.

In their abstract, Renella, Orkin, Williams and their colleagues reported on their efforts to develop a combined lentiviral gene transfer/RNA interference approach capable of turning down BCL11A in vitro in human and mouse cells and in vivo in a mouse bone-marrow transplant model. Among their findings, the researchers documented a five- to 20-fold increase in fetal hemoglobin production in treated mice.

The team is quick to note that they have not yet attempted to correct disease in a model of sickle cell using this approach. Such efforts would be carried out in future studies.

"There are many approaches to sickle cell disease, and while we've shown we can develop viral vectors, knock down BCL11A, and induce fetal hemoglobin production, it's all been done in non-disease model systems," cautioned Renella, a fellow in the Williams laboratory at DF/CHCC. "What we don't yet know is whether we can stop production of sickled cells in a sickle cell system. Further experiments will show us if that is possible."


'/>"/>

Contact: Rob Graham
rob.graham@childrens.harvard.edu
617-919-3110
Boston Children's Hospital
Source:Eurekalert

Related medicine news :

1. Targeted prostate biopsy has potential to improve diagnosis of prostate cancer
2. Study identifies potential new pathway for drug development
3. US Drug Watchdog Expands Their Transvaginal Mesh Initiative Because Of The Potential Number Of Victims & They are Offering the Names of the Best Women Attorneys To Help
4. Many home couches contain potentially toxic flame retardants
5. Scripps Research Institute study points to potential new therapies for cancer and other diseases
6. A*STAR scientists identify potential drug target for inflammatory diseases including cancers
7. Researchers report potential new treatment to stop Alzheimers disease
8. ACA: More than a million women could gain access to potentially life saving tests for cancer
9. RSV study shows potential for vaccine strategies to protect babies
10. Potential new technique for anticancer radiotherapy could provide alternative to brachytherapy
11. Micro-Stents May Hold Potential to Treat Glaucoma
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/24/2017)... ... March 24, 2017 , ... ... contaminated soil, dredged material, and hazardous and non-hazardous materials announced today the acquisition ... This acquisition will add four additional processing facilities and a vast array ...
(Date:3/24/2017)... ... March 24, 2017 , ... The Radiology Business Management Association ... The annual awards, now in their 12th year, are among the most prestigious in ... In 2016, the awards were retooled to recognize achievements in both large budget (over ...
(Date:3/24/2017)... ... March 24, 2017 , ... Shamanic healer and teacher ... Healing and Spiritual Awakening, proudly presents her Sacred Peru retreat with world ... and spiritual journey during the Summer Solstice will also be her final international ...
(Date:3/24/2017)... ... ... The law firm of Enea, Scanlan & Sirignano, LLP, with ... C. Enea has joined the firm as an associate attorney. Ms. Enea, who previously ... law, Medicaid planning and applications, and Wills, Trusts and Estates. Samantha A. Lyons, ...
(Date:3/24/2017)... ... March 24, 2017 , ... “Finding ... God #TruthwithGrace”: a devotional journal chronicling the writer’s path toward true communion with ... Walk with God #TruthwithGrace” is the creation of published author Lea Michelle Johnson, ...
Breaking Medicine News(10 mins):
(Date:3/27/2017)... 2017  PhaseRx, Inc. (NASDAQ: PZRX), a biopharmaceutical ... diseases in children, today reported financial results for ... 31, 2016 and provided an update on its ... make progress during the fourth quarter of 2016 ... primate safety study, and with our lead development ...
(Date:3/24/2017)... , March 24, 2017 Abdominal Aortic Aneurysm Repair Devices ... by 2022, Globally, registering a CAGR of 5.1% from 2016 to 2022. The endovascular ... to dominate the market during the study period. Continue ... ... ...
(Date:3/24/2017)... , Mar. 24, 2017 Research ... Management in the U.S.: Consumer Strategies" report to their ... ... how adults approach and treat their physical pain, emphasizing consumer ... distinct groups: pain sufferers and adults who have selected illnesses/conditions ...
Breaking Medicine Technology: