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:8/3/2015)... Calif. , Aug. 3, 2015 Synaptics, ... human interface solutions, today announced that members of the ... at the Pacific Crest Global Technology Leadership Forum on ... The conference will be held at the Sonnenalp Resort ... The presentation may include forward-looking information. An audio ...
(Date:7/31/2015)... 2015 Den 10. internationale konference om Genomik ... 22. - 25. oktober i Shenzhen ... fødselsdag i år. Siden starten i 2006 er ICB ... ,omik,-feltet, og er en af de mest dynamiske, entusiastiske ... på nylige gennembrud og fremskridt inden for ,omik,-forskning, som ...
(Date:7/31/2015)... , 31 de julio de 2015 BGI llevará ... www.icg-10.org ) del 22 al 25 de octubre de ... La conferencia celebra su décimo aniversario este año. ... en una de las reuniones anuales más influyentes del ... una de las conferencias científicas más dinámicas, entusiastas y ...
Breaking Biology News(10 mins):Den 10. internationale konference om Genomics (ICG-10) åbner i oktober 2La 10ª Conferencia Internacional sobre Genómica (ICG-10) comienza en octubre 2La 10ª Conferencia Internacional sobre Genómica (ICG-10) comienza en octubre 3
... volume of online scientific data, a new style of ... have a direct impact on scientists and clinicians. ... Biology and Faculty of 1000 Medicine, are each launching ... journals, called F1000 Reports, are unlike existing scientific journals ...
... wide-open spaces of the Flint Hills may no longer ... familiar grassland birds, according to research by a Kansas ... found that three bird species common to the Flint ... population decline in the face of extensive land-management practices ...
... epidemiological study of men and post-menopausal women primarily over ... associated with greater bone mineral density (BMD). Researchers at ... Aging (USDA HNRCA) at Tufts University found associations were ... significantly lower in men drinking more than two servings ...
Cached Biology News:World's experts identify the hottest trends in biology and medicine 2Birds in Flint Hills of Kansas, Oklahoma face population decline despite large habitat 2Birds in Flint Hills of Kansas, Oklahoma face population decline despite large habitat 3Moderate alcohol intake associated with bone protection 2Moderate alcohol intake associated with bone protection 3
(Date:8/3/2015)...  Halozyme Therapeutics, Inc. (NASDAQ: HALO ) will ... quarter 2015 on Monday, August 10, 2015 at 4:30 ... president and chief executive officer, will lead the call. ... results for the second quarter 2015. The ... of Halozyme,s corporate website and a recording will be ...
(Date:8/3/2015)... 2015  ArroGen Group, an integrated advanced forensic ... Identification™ (FMID) technology Aug. 2-8 at the International ... Sacramento, Calif. FMID, a ... enforcement agents, district attorneys and government agencies to ... By analyzing chemical residues on fingerprints taken ...
(Date:8/3/2015)... At the 11th hour, London,s ... of Jude Liversage,s campaign so the seven-year-old ...      (Photo: http://photos.prnewswire.com/prnh/20150803/254228 ) ... The Selective Dorsal Rhizotomy (SDR) procedure reduced the spasticity ... wish to kick a ball around like his schoolmates. ...
(Date:8/3/2015)... Ga. , Aug. 3, 2015  MiMedx Group, ... company utilizing human amniotic tissue and patent-protected processes to ... Wound Care, Surgical, Orthopedic, Spine, Sports Medicine, Ophthalmic and ... present at the 35 th Annual Canaccord|Genuity Growth ... H. "Pete" Petit, Chairman and CEO, William C. ...
Breaking Biology Technology:Halozyme to Host Second Quarter 2015 Financial Results Conference Call 2ArroGen Previews New Fingerprint Molecular Identification Technology 2Cerebral Palsy Operation by The Portland Hospital Allows a Boy to Stand Properly for the First Time 2Cerebral Palsy Operation by The Portland Hospital Allows a Boy to Stand Properly for the First Time 3MiMedx To Present At 35th Annual Canaccord|Genuity Growth Conference 2
... Berkley Life Sciences, LLC, a,member company of ... LS Prime. LS Prime is a uniquely innovative ... faced by life,science companies of all sizes., ... errors and omissions, and premises operations coverages. Included,with ...
... ... Radiesse, SAN MATEO, Calif., June 26 BioForm Medical,Inc. ... studies in the,June 2008 issue of the Journal of Dermatologic ... The five,published clinical and preclinical reports were:, -- A ...
... 26 /Xinhua-PRNewswire-FirstCall/ --,China-Biotics, Inc. (OTC Bulletin ... Chinese firm specializing in the manufacture,research, ... products,today announced it signed a one-year ... ("Lijia") to supply probiotics for Lijia,s,probiotics-based ...
Cached Biology Technology:Berkley Life Sciences Introduces LS Prime: A Revolutionary New Primary Liability Policy for the Life Science Industry 2Radiesse(R) Dermal Filler Featured Prominently in Journal of Dermatologic Surgery Special Issue on Fillers 2Radiesse(R) Dermal Filler Featured Prominently in Journal of Dermatologic Surgery Special Issue on Fillers 3Radiesse(R) Dermal Filler Featured Prominently in Journal of Dermatologic Surgery Special Issue on Fillers 4Radiesse(R) Dermal Filler Featured Prominently in Journal of Dermatologic Surgery Special Issue on Fillers 5Radiesse(R) Dermal Filler Featured Prominently in Journal of Dermatologic Surgery Special Issue on Fillers 6China-Biotics, Inc. to Supply Probiotics to Pharmaceutical Company 2China-Biotics, Inc. to Supply Probiotics to Pharmaceutical Company 3
... Complete Solution Polymorphic DNA ... We provide a complete solution for SNP ... assay design, oligonucleotide primers, PCR amplification, DNA ... For SNP discovery, Polymorphic uses ...
... mini ReadySub-Cell GT cell and PowerPac basic power ... of nucleic acids in agarose gels. The mini ... cables and electrodes, and leveling bubble. The PowerPac ... output of 10-300 V, 4-400 mA, and 75 ...
... CE-based system provides clear advantages over ... load, gene expression), mutation analysis, and ... system with microplate connectivity is ideal ... quality control. The P/ACE Series ...
The imaging screen-K (Kodak), tritium, is a 20 x 25 cm storage phosphor screen that is used for the quantitation and imaging of 32P, 33P, 35S, 14C, and 3H....
Biology Products: