Navigation Links
Children's Hospital of Philadelphia collaborates in gene therapy success in an immune disorder
Date:11/15/2010

A pediatric immunologist at The Children's Hospital of Philadelphia collaborated with European gene therapy researchers who achieved marked clinical improvements in two young children with Wiskott-Aldrich syndrome, a very rare but often severe immunodeficiency disorder.

Jordan S. Orange, M.D., Ph.D., an expert in Wiskott-Aldrich syndrome (WAS), performed sophisticated cell imaging and analysis for the study, led by German researcher Christoph Klein, M.D., Ph.D., of Hannover Medical School. Orange was a senior co-author, and Children's Hospital was the only U.S. institution represented in the study, published in the Nov. 11 New England Journal of Medicine.

The proof-of-principle study marks the latest example of a recent upswing in clinical success for gene therapy. In 2009, researchers from The Children's Hospital of Philadelphia and the University of Pennsylvania reported dramatic vision improvements in patients with Leber congenital amaurosis (LCA), a form of inherited blindness. In the same year, Parisian researchers announced success in treating adrenoleukodystrophy, the disease depicted in the movie "Lorenzo's Oil." And scientists at the University of California are reporting preliminary clinical benefits in gene therapy for adenosine deaminase deficiency, an immune deficiency disorder related to "bubble boy disease."

Like WAS, all are very rare inherited disorders and have the potential to be severe and (except for LCA) life-threatening.

Wiskott-Aldrich syndrome is a complex X-linked immunodeficiency disorder characterized by recurrent infections, eczema and thrombocytopeniaa low platelet count. Mutations in the WAS gene disable its ability to produce WAS protein, which plays a crucial role in different types of immune cells. Without WAS protein, immune cells are disabled, incapable of providing immune defenses. Hence, patients are susceptible to premature death from infection, cancers or bleeding.

Alt
'/>"/>

Contact: John Ascenzi
Ascenzi@email.chop.edu
267-426-6055
Children's Hospital of Philadelphia
Source:Eurekalert  

Page: 1 2 3

Related biology news :

1. Childrens Hospital LA discovers growth factor essential to epicardial cell function
2. Brown University and Women & Infants Hospital expand national childrens study to Bristol County
3. Field-hospital-on-a-chip project awarded to nanoengineer from UC San Diego
4. First results from hospital trials testing
5. Rhode Island Hospital simulation center examines benefits and applications of medical simulation
6. Childrens Hospital scientists achieve repair of injured heart muscle in lab tests of stem cells
7. International copper industry defines role in the fight against hospital infections
8. Mount Sinai Hospital researcher makes stem cell breakthrough
9. Research could lead to new non-antibiotic drugs to counter hospital infections
10. Childrens Hospital Oakland is first pediatric hospital to use laboratory-on-a-card technology
11. Childrens Hospital Oakland scientists first to discover new source for harvesting stem cells
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Children's Hospital of Philadelphia collaborates in gene therapy success in an immune disorder
(Date:9/12/2014)... attack from a pathogen, they sometimes respond by killing ... committing "cell suicide" to thwart further damage from the ... microscopic, spots or lesions on the plant. , But ... the plant regulates this "spotty" defense mechanism because the ... North Carolina State University have identified a number of ...
(Date:9/12/2014)... of dendritic cells in human skin have assorted functions ... report researchers in the journal EMBO Molecular Medicine ... the composition of dendritic cells in psoriatic skin lesions ... the disease. , "We urgently need new ways ... to patients and reduce the incidence of known side ...
(Date:9/11/2014)... Northumberland and Durham" by Nathaniel John Winch is re-published ... an example of combining modern information technology together with ... and data re-use. This publication will be supporting ongoing ... be seen as a model for other regions in ... the meticulous detail of old books with the interconnectedness ...
Breaking Biology News(10 mins):Corn spots: Study finds important genes in defense response 2Dendritic cells affect onset and progress of psoriasis 2Re-publication of 'Flora of Northumberland and Durham' (1831): A dramatic account of change 2Re-publication of 'Flora of Northumberland and Durham' (1831): A dramatic account of change 3
... ecosystem below an Antarctic glacier and learned that it survived ... growth. Described in the April 17 issue of ... a pool of brine trapped below Taylor Glacier and next ... Priscu , co-author of the Science article. Priscu ...
... over, Y chromosome it,s time X got some ... chromosome associated with being female, University of California, Berkeley, ... history of the X chromosome is every bit as ... important clues to the origins and benefits of sexual ...
... Sven Enerbck, Professor at the Institute of Biomedicine at the ... scientists who published their results in The New England ... Enerbck and others show that adults use brown fat to ... new possibilities in treating overweight and obesity. It has ...
Cached Biology News:Ancient ecosystem thrives millions of years below Antarctic glacier 2The story of X -- evolution of a sex chromosome 2The story of X -- evolution of a sex chromosome 3The story of X -- evolution of a sex chromosome 4
(Date:9/12/2014)... (PRWEB) September 12, 2014 Advancing ... and decontamination of chemical and biological threats is ... by Worcester Polytechnic Institute (WPI) and the Georgian ... the Science for Peace and Security Programme of ... of NATO’s Advanced Research Workshop series, the event ...
(Date:9/12/2014)... (PRWEB) September 12, 2014 Today, “The ... on Indiegogo. , The film, directed by former ... Aldrin, Shimon Peres, Ashton Kutcher, Will.i.Am, Michio Kaku, and ... and there’s only one university in the world devoted ... change the lives of a billion people: Singularity University. ...
(Date:9/12/2014)... 2014 Sandia Biotech, Inc. announced today ... with STC.UNM (STC) for a technology developed by researchers ... New Mexico, and Queen Mary University of London in ... Recombinant Fluorescent Antibody Technology Platform (RFAP) was developed by ... and Dr. Angray Kang from Queen Mary’s Department of ...
(Date:9/11/2014)... (hiPSCs), researchers have gained new insight into what ... of neuronal signaling associated with this disease. They ... hiPSCs of healthy individuals and of patients with ... cells to release neurotransmitters, such as dopamine, that ... to various disorders. By discovering a simple method ...
Breaking Biology Technology:Worcester Polytechnic Institute to Lead Scientific Workshop on Nanoscale Defenses for Biological and Chemical Threats 2Worcester Polytechnic Institute to Lead Scientific Workshop on Nanoscale Defenses for Biological and Chemical Threats 3Documentary Film that Follows Singularity University Students as They Change the World Launches its IndieGoGo Fundraising Campaign 2Sandia Biotech and STC.UNM Announce Option Agreement to License Novel Recombinant Antibody Technology 2Sandia Biotech and STC.UNM Announce Option Agreement to License Novel Recombinant Antibody Technology 3Sandia Biotech and STC.UNM Announce Option Agreement to License Novel Recombinant Antibody Technology 4Stem cells help researchers understand how schizophrenic brains function 2
... 16 Monogram,Biosciences, Inc. (Nasdaq: MGRM ) ... of the company,s HIV tropism assay were presented ... Drug Resistance Workshop,in Sitges, Spain. Trofile was introduced ... in class CCR5 antagonist, in,August 2007. Recent enhancements ...
... /PRNewswire-FirstCall/ - ProMetic Life Sciences Inc.,(TSX: PLI) ... compound in clinical trial in patients with ... the European Hematology Association,held in Copenhagen, Denmark ... Director R&D, Biology, presented data on,PBI-1402 in ...
... Inc. (Nasdaq: ALXN ) today announced that ... of Alexion,Pharmaceuticals, will present at the William Blair ... a.m., local time (10:10 a.m., eastern) on,Thursday, June ... Sinha,s remarks will be available live. You can,access ...
Cached Biology Technology:Sensitivity Enhancements to Monogram's Trofile(TM) Assay Demonstrate Improved Power to Select Patients for CCR5 Antagonist Therapy 2Sensitivity Enhancements to Monogram's Trofile(TM) Assay Demonstrate Improved Power to Select Patients for CCR5 Antagonist Therapy 3Sensitivity Enhancements to Monogram's Trofile(TM) Assay Demonstrate Improved Power to Select Patients for CCR5 Antagonist Therapy 4Sensitivity Enhancements to Monogram's Trofile(TM) Assay Demonstrate Improved Power to Select Patients for CCR5 Antagonist Therapy 5Sensitivity Enhancements to Monogram's Trofile(TM) Assay Demonstrate Improved Power to Select Patients for CCR5 Antagonist Therapy 6Positive clinical data for PBI-1402 presented at the Annual Congress of the European Hematology Association 2Positive clinical data for PBI-1402 presented at the Annual Congress of the European Hematology Association 3
...
... Lyophilized. Lyophilized from 2 mM sodium ... human liver. Cathepsin D is a major ... as a 52 kDa proenzyme. Overexpression of ... associated with higher risk of relapse and ...
... Grade Thrombin is a highly purified preparation ... with appropriate vectors. The preparation is functionally ... is free of detectable contaminating proteases. Prepared ... by certified tests to be negative for ...
Rat Hepatocytes (RH) (>3,000,000 cells)...
Biology Products: