Navigation Links
Potential gene therapy for Sickle cell disease
Date:7/2/2013

Sickle cell disease (SCD) is an autosomal recessive disorder caused by mutations in hemoglobin (HBB) that deform red blood cells. A small number of patients have been successfully treated with allogeneic hematopoietic stem cell (HSC) transplantation; however, there are several drawbacks and complications associated with this procedure, including graft vs. host disease and long-term immune suppression. Many of complications could potentially be avoided by the use of autologous HSC transplant, which uses patient cells that have been treated genetically modified to replace defective hemoglobin. In this issue of the Journal of Clinical Investigation, Zulema Romero and colleagues investigated the utility of a genetic vector encoding a human hemoglobin gene engineered to impede sickle hemoglobin and prevent the sickling of red blood cells. The vector was used to efficiently transduce bone marrow cells from SCD patients and the transduced cells were successfully transplanted into immunocompromised mice, suggesting that this method could potentially be used to treat SCD.


'/>"/>

Contact: Jillian Hurst
press_releases@the-jci.org
Journal of Clinical Investigation
Source:Eurekalert

Page: 1

Related biology technology :

1. Dual-acting class of antimalarial compounds discovered with potential to prevent and treat malaria
2. The Pharmabiotic Research Institute (PRI) is Preparing to Publish its First Works on the Therapeutic and Medicinal Potential of Probiotics
3. Carnegie Mellon study reveals potential of manganese in neutralizing deadly Shiga toxin
4. GeoVax Labs Presents New Findings on Potential HIV Vaccine at 2012 Conference on Retroviruses and Opportunistic Infections (CROI)
5. Carbon capture and storage -- new research from UKERC shows tough road ahead to realize potential
6. Researchers Find Potential Link Between Drinking Alcohol And Breast Cancer
7. Golden potential for gold thin films
8. Exosome Diagnostics Presents Data at American Urology Association Demonstrating Potential Utility of Urine Exosomes to Non-invasively Detect and Manage Prostate Cancer
9. Rising Interest of Big Pharma Creates Immense Growth Potential for Contract Manufacturing Organisations, Says Frost & Sullivan
10. Welcome to BIO-Poland Explore Our Potential
11. AstraZeneca and Rigel Sign Worldwide License Agreement for a Potential New Treatment for Chronic Asthma
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/21/2014)... November 21, 2014 2014 Deep ... Industryā€¯ is a professional and in-depth research report ... ferrocyanide information, like its definition, classification, application, ... overview. This report covers the international market analysis, ... (such as the US, Europe, Asia, China, Japan ...
(Date:11/21/2014)... , Nov. 20, 2014 /CNW/ - Aequus Pharmaceuticals ... announce that it has closed a brokered private ... for approximately C$3.7 million.  Cormark Securities Inc. and ... the Brokered Offering for a syndicate of agents ... Financial Corp. (collectively the "Agents"). Concurrently with the ...
(Date:11/18/2014)... SAN BRUNO, Calif. , Nov. 18, 2014 ... is a platinum sponsor of the 10th Annual World Stem ... and regenerative medicine stakeholders. The 2014 World Stem Cell Summit ... San Antonio, Texas . ... the discovery and development of lifesaving cures and therapies, convening ...
(Date:11/18/2014)... pH measurement and control is vital in a ... food and beverage industries. But what exactly is pH, ... sensors work? METTLER TOLEDO has produced a guide to ... these questions and more. , METTLER TOLEDO provides a ... The actual sensor, the pH electrode, is therefore the ...
Breaking Biology Technology:Sodium Ferrocyanide Market: Analysis for Global & China Regions Development Trends Available at DeepResearchReports.com 2Sodium Ferrocyanide Market: Analysis for Global & China Regions Development Trends Available at DeepResearchReports.com 3Aequus Pharmaceuticals closes $4.2 million private placement financing 2Aequus Pharmaceuticals closes $4.2 million private placement financing 3Cord Blood Registry Sponsors 2014 World Stem Cell Summit 2Cord Blood Registry Sponsors 2014 World Stem Cell Summit 3pH Guide from METTLER TOLEDO Explains Measurement, Theory and Practice of pH Applications 2
... Group Teams Up with YWCA & Roche to Educate ... of Balancing Cancer and a Career -, NEW ... Month, Cancer and Careers, a non-profit program,founded by Cosmetic ... offers guidance for the hundreds of thousands of people,living ...
... Calif., March 10 Accuray,Incorporated (Nasdaq: ARAY ), ... that its management is scheduled to present at the ... Seasons Hotel in,Las Vegas on Wednesday, March 19, 2008 ... live webcast of the presentation will be available online ...
... SAN FRANCISCO, Calif., March 10 ,Anesiva, Inc. (Nasdaq: ... has submitted,a supplemental New Drug Application (sNDA) with ... the indication for Zingo to treat the pain,associated ... adults.,Zingo(TM) (lidocaine hydrochloride monohydrate) powder intradermal,injection system is ...
Cached Biology Technology:First-Ever 'Cancer and Careers' Seminar Debuts in Orange County 2First-Ever 'Cancer and Careers' Seminar Debuts in Orange County 3Accuray to Present at Citi Investment Research Small & Mid-Cap Conference 2Anesiva Announces Submission of Supplemental New Drug Application for Zingo(TM) to Reduce Pain Associated With Needle Insertion Procedures in Adults 2Anesiva Announces Submission of Supplemental New Drug Application for Zingo(TM) to Reduce Pain Associated With Needle Insertion Procedures in Adults 3Anesiva Announces Submission of Supplemental New Drug Application for Zingo(TM) to Reduce Pain Associated With Needle Insertion Procedures in Adults 4
(Date:11/12/2014)... Nov. 12, 2014 Crossmatch™, a leading provider ... U.are.U ® fingerprint readers have been deployed throughout ... Central Mexico . The bakery chain implemented ... payroll issues caused by employees clocking in for each ... fingerprint readers, Montparnasse relied on paper timecards and a ...
(Date:11/7/2014)... SPRINGS, Florida , November 6, 2014 ... secure authentication for better mobile security revolutionizing online transactions.  Companies ... ), Alibaba Holdings Ltd. (NYSE: BABA ), Google ... MSFT ), eBay Inc. (NASDAQ: EBAY ) ... NXTD and NXTDW), a biometric authentication company ...
(Date:11/5/2014)... in the UTSA College of Sciences, is one ... receive a two-year $300,00 National Science Foundation (NSF) ... funding supports President Obama,s BRAIN Initiative, a federal ... that will demystify complex brain processes. , ... broken into the interactions of multiple components, each ...
Breaking Biology News(10 mins):Montparnasse Pasteleria Achieves Time and Attendance Transparency with Crossmatch U.are.U Fingerprint Readers 2Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 2Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 3Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 4Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 5Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 6UTSA biology professor awarded $300,000 NSF grant for brain research 2UTSA biology professor awarded $300,000 NSF grant for brain research 3
... team of Fox Chase Cancer Center scientists led by ... mutation that keeps a mouse strain from developing white ... report by Kappes and his colleagues appears in the ... discovered the mice with this naturally occurring defect in ...
... simian virus 40 produces the Large T antigen which inactivates ... pRb. In a study published in the Journal of Biological ... the discovery of an additional target for T antigen--a protein ... chromosomal region that is deleted in up to 30% of ...
... demonstrated that a gel applied in the vagina provides ... the herpes simplex Virus. The study, presented at the ... first to show that a gel can retain anti-viral ... was funded by the National Institutes of Health (NIH) ...
Cached Biology News:Fox Chase Cancer Center scientists identify immune-system mutation 2New binding target for oncogenic viral protein 2Topical treatment shown to inhibit HIV and herpes simplex virus infection 2
... and lightweight, the Moticam 2300 camera (MC2300) ... eyepiece, eyetube, or trinocular port. Included in ... Plug-and-Play Images 2.0ML software offers quick and ... to maximize resolution for examination or documentation ...
... Physical form: Solution in 50% glycerol containing 20 mM ... and 20 mM NaCl Preparation Unit Definition: One unit ... equivalent to a ΔA 260 of 1.0 in ... volume 2.625 ml). Assay: SDS-PAGE ≥ 90 % ...
... an aqueous solution that contains a purified ... a PBS buffer (phosphate buffered saline), pH ... not contain a preservative. StabilCoat Immunoassay ... dried protein components in immunoassays and can ...
... Stabilizer is a bovine protein-free alternative ... is an aqueous solution that contains ... buffer (3-(N-Morpholino) propanesulfonic acid), pH 6.0 ... of 0.02% methylisothiazolone and 0.02% bromonitrodioxane ...
Biology Products: