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:2/11/2016)... Feb. 11, 2016  Wellcentive today announced it ... Portland, Oregon -based community care organization (CCO) ... health analytics, quality reporting and care management solutions ... its team of quality managers, analysts and care ... provider groups serving FamilyCare members. ...
(Date:2/11/2016)... BEACH, Florida , February 11, 2016 /PRNewswire/ ... PositiveID Corporation ("PositiveID" or "Company") (OTCQB: PSID), ... diagnostics, announced today that its Thermomedics subsidiary, which ... progress on its growth plan in January 2016, ... products distributors, increasing sequential monthly sales growth, and ...
(Date:2/11/2016)... ... , ... Global Stem Cells Group, a world ... Cells Network (GSCN) to distribute exosome injection and other biological products to stem ... Dominican Republic, Colombia, Argentina, Nicaragua, Panama, El Salvador, Venezuela, Peru, Ecuador, Paraguay, Puerto ...
(Date:2/10/2016)... Early-career researchers from Indonesia ... Uganda and Yemen ... Indonesia , Nepal , ... Yemen are being honored for their accomplishments in nutrition, psychiatry, ... mentoring young women scientists who are pursuing careers in agriculture, biology and ...
Breaking Biology Technology:
(Date:1/21/2016)... , January 21, 2016 ... a new market research report "Emotion Detection and Recognition ... Software Tools (Facial Expression, Voice Recognition and Others), ... Global forecast to 2020", published by MarketsandMarkets, the ... to reach USD 22.65 Billion by 2020, at ...
(Date:1/18/2016)... Calif. , Jan. 18, 2016  Extenua ... software that simplifies the use and access of ... and go-to-market partnership with American Cyber.  ... brings extensive experience leading transformational C4ISR and Cyber ... and integrating the latest proven technology solutions," said ...
(Date:1/11/2016)... , Jan. 11, 2016 Synaptics Incorporated (NASDAQ: ... solutions, today announced that its ClearPad ® TouchView ... products won two separate categories in the 8 th ... and Best Technology Breakthrough. The Synaptics ® TDDI ... simplified supply chain, thinner devices, brighter displays and borderless ...
Breaking Biology News(10 mins):