Navigation Links
Sickle cell disease corrected in human models using stem cell-based gene therapy

own cure other than stem cell transplantation.

To treat SCD, Sloan-Kettering scientists devised a novel engineering strategy combining RNA interference with globin gene transfer by creating a therapeutic transgene, consisting of the gamma-globin gene and small interfering RNA specific for beta S-globin, the globin mutant chain that causes sickle cell disease.

"An innovative and sophisticated approach was needed to genetically engineer hematopoietic stem cells using a practical and clinically applicable process. The transferred gene must not disrupt the cells' normal functions," explained Isabelle Riviere, PhD, Co-Director of the Gene Transfer and Somatic Cell Engineering Facility and a study co-author.

The new gene had two functions -- produce normal hemoglobin and suppress the generation of sickle shaped hemoglobin S. The therapeutic gene was engineered into a lentiviral vector and introduced into hematopoietic stem cells. After the cells received the treatment, they made normal hemoglobin.

"This proved our hypothesis that you can simultaneously add one function and delete another in the same cell and obtain synergistic genetic modifications within a single cell," said Selda Samakoglu, PhD, a member of Dr. Sadelain's laboratory and the study's first author. "In this case, we used the technique to correct sickle cell disease, but it should be broadly applicable to use therapeutically in stem cells or malignant cells."


'"/>

Source:Memorial Sloan-Kettering Cancer Center


Page: 1 2

Related biology news :

1. Discovery Promises Simpler Therapy for Sickle Cell Disease
2. Sickle cell and protection against malaria
3. Neuronal traffic jam marks early Alzheimers disease
4. Scientists detect probable genetic cause of some Parkinsons disease cases
5. Ecological destruction fuels emerging diseases
6. Newly discovered virus linked to childhood lung disorders and Kawasaki disease
7. U of M researcher examines newly emerging deadly disease
8. Marburg virus disease in Angola - update
9. Molecular machine may lead to new drugs to combat human diseases
10. Female sex hormones play a vital role in defense against sexually transmitted diseases
11. A bacterial genome reveals new targets to combat infectious disease
Post Your Comments:
(Date:3/19/2015)... March 19, 2015  NXT-ID, Inc. (NASDAQ: NXTD ... on the growing mobile commerce market, announces its biometric payment ... clip that aired this week on Washington DC,s ... In a segment "The Next Great Thing", host ... way to pay, and ,a really big breakthrough in mobile ...
(Date:3/17/2015)... MINNETONKA, Minn. , March 17, 2015 /PRNewswire/ ... sciences technology company specializing in clinical study management ... will be in attendance at ARENA,s Outsourcing in ... Cary, NC.  This event provides an excellent forum ... company,s easy-to-use, flexible and affordable software-as-a-service (SaaS) eClinical ...
(Date:3/17/2015)... March 17, 2015  MecklerMedia Corporation (OTCQX: MECK) ... for service robots ever held in ... on May 11-13, 2015 at the Javits Convention ... the event include: Acorn Product Development; Axis NJ; ... Kinova Robotics; Littler; NewBotic Corporation; Neya Systems LLC; ...
Breaking Biology News(10 mins):NXT-ID's Wocket Smart Wallet Featured on Fox News Segment, "The Next Great Thing" 2NXT-ID's Wocket Smart Wallet Featured on Fox News Segment, "The Next Great Thing" 3Connect With MedNet Solutions At Outsourcing In Clinical Trials Southeast 2MecklerMedia's RoboUniverse New York Announces Sponsors and Exhibitors, May 11-13, 2015 2
... is a severely debilitating psychiatric disease that is thought ... nervous system; however, major breakthroughs linking its genetics to ... PhD assistant professor of Pediatrics at Northwestern University,s Feinberg ... Molecular Genetics Program of Children,s Memorial Research Center studies ...
... A common variation of the gene involved in ... linked to problem behaviors in adults with developmental and ... published in the July 2009 issue of the ... are available online at http://tinyurl.com/mw8baj . "Problem ...
... only a few millimeters up to a few centimeters in ... research, because they possess the extraordinary ability to regenerate lost ... even grow an entirely new worm out of minute amputated ... Berlin, Germany together with researchers in the US and Canada ...
Cached Biology News:A genetic basis for schizophrenia 2Genetic marker linked to problem behaviors in adults with developmental disabilities 2International research team seeks to unravel flatworm regeneration 2
(Date:3/27/2015)... YORK , March 27, 2015  Peter Walter ... Biomedical Science for his pioneering work on how proteins ... components of a regulatory mechanism that cells use to ... Dr. Walter is a professor of biochemistry at the ... groundwork for treating a range of human diseases related ...
(Date:3/26/2015)... March 26, 2015  Roka Bioscience, Inc. (NASDAQ: ... on providing advanced testing solutions for the detection ... for the three months and full year ended ... Financial Results:Revenue for the quarter ended December 31, ... the fourth quarter of 2013 and $1.5 million ...
(Date:3/26/2015)... and CINCINNATI , March ... (LSE: SHP, NASDAQ: SHPG ) and Cincinnati ... broad research collaboration for rare diseases. The goal of ... therapies to treat rare diseases with high unmet ... with Cincinnati Children,s research expertise. As a nationally ...
(Date:3/26/2015)... Ale Gicqueau, Founder and President of Clinovo, will ... Align Technology, on “ Conducting Postmarket Registries - Rationale ... Conference and Exhibition on April 25th through April 28th ... poster presentation, Ale Gicqueau and Victor Chen will discuss ... the requirements and resources to conduct the registry, as ...
Breaking Biology Technology:German-born cell biologist Peter Walter wins the 2015 Vilcek Prize in Biomedical Science 2German-born cell biologist Peter Walter wins the 2015 Vilcek Prize in Biomedical Science 3Roka Bioscience Reports Fourth Quarter and Full Year 2014 Financial Results 2Roka Bioscience Reports Fourth Quarter and Full Year 2014 Financial Results 3Roka Bioscience Reports Fourth Quarter and Full Year 2014 Financial Results 4Roka Bioscience Reports Fourth Quarter and Full Year 2014 Financial Results 5Roka Bioscience Reports Fourth Quarter and Full Year 2014 Financial Results 6Roka Bioscience Reports Fourth Quarter and Full Year 2014 Financial Results 7Shire and Cincinnati Children's Establish Rare Disease Research Collaboration 2Shire and Cincinnati Children's Establish Rare Disease Research Collaboration 3Shire and Cincinnati Children's Establish Rare Disease Research Collaboration 4Shire and Cincinnati Children's Establish Rare Disease Research Collaboration 5Clinovo Presents at ACRP 2015 in Salt Lake City, Utah on April 25th-28th 2Clinovo Presents at ACRP 2015 in Salt Lake City, Utah on April 25th-28th 3
... Advanced Scientific Knowledge, Highlight Most Recent ... ... ArcMesa Educators, an accredited,provider of continuing education courses, is proud ... by years of scientific,experience and advanced degrees in molecular biology, structural ...
... AUSTIN, Texas, Sept. 27 Luminex Corporation,(Nasdaq: ... at the,Biotechnology Industry Organization Investor Forum to be ... investor presentation by Patrick J. Balthrop, president and ... officer, will,begin at 9:30 a.m. Pacific time; 12:30 ...
... GENZ ) confirmed today that it will not change ... $5.60 in cash per,outstanding common share as set forth ... owns approximately 22 percent of the,outstanding shares of Bioenvision ... its preferred stock that carry a unique set of,rights ...
Cached Biology Technology:ArcMesa Educators Appoints Medical Advisor and Directors to CME Development Team 2ArcMesa Educators Appoints Medical Advisor and Directors to CME Development Team 3Luminex Corporation to Present at Biotechnology Industry Organization Investor Forum 2Genzyme Affirms Purchase Price for Bioenvision at $5.60 Per Outstanding Share 2Genzyme Affirms Purchase Price for Bioenvision at $5.60 Per Outstanding Share 3
... Nonanoyl-N-methylglucamide White solid. ... be readily removed by dialysis. Useful ... ≥98% by HPLC. Absorbance (10%, ... 2 O. CMC 19 - 25 ...
...
Mouse polyclonal antibody to TNPO2 - transportin 2 (importin 3, karyopherin beta 2b)...
... Rabbit polyclonal to HDAC4 - ChIP ... the class II mammalian histone deacetylases, which ... C-terminal sequence is highly similar to the ... other deacetylases, shuttles between the nucleus and ...
Biology Products: