Navigation Links
Combined stem cell-gene therapy approach cures human genetic disease in vitro
Date:5/31/2009

LA JOLLA, CAA study led by researchers at the Salk Institute for Biological Studies, has catapulted the field of regenerative medicine significantly forward, proving in principle that a human genetic disease can be cured using a combination of gene therapy and induced pluripotent stem (iPS) cell technology. The study, published in the May 31, 2009 early online edition of Nature, is a major milestone on the path from the laboratory to the clinic.

"It's been ten years since human stem cells were first cultured in a Petri dish," says the study's leader Juan-Carlos Izpisa Belmonte, Ph.D., a professor in the Gene Expression Laboratory and director of the Center of Regenerative Medicine in Barcelona (CMRB), Spain. "The hope in the field has always been that we'll be able to correct a disease genetically and then make iPS cells that differentiate into the type of tissue where the disease is manifested and bring it to clinic."

Although several studies have demonstrated the efficacy of the approach in mice, its feasibility in humans had not been established. The Salk study offers the first proof that this technology can work in human cells.

Belmonte's team, working with Salk colleague Inder Verma, Ph.D., a professor in the Laboratory of Genetics, and colleagues at the CMRB, and the CIEMAT in Madrid, Spain, decided to focus on Fanconi anemia (FA), a genetic disorder responsible for a series of hematological abnormalities that impair the body's ability to fight infection, deliver oxygen, and clot blood. Caused by mutations in one of 13 Fanconi anemia (FA) genes, the disease often leads to bone marrow failure, leukemia, and other cancers. Even after receiving bone marrow transplants to correct the hematological problems, patients remain at high risk of developing cancer and other serious health conditions.

After taking hair or skin cells from patients with Fanconi anemia, the investigators corrected the defective gene in the
'/>"/>

Contact: Gina Kirchweger
Kirchweger@salk.edu
858-453-410-01340
Salk Institute
Source:Eurekalert  

Page: 1 2

Related biology news :

1. Extended wakefulness, combined with alcohol, severely impairs driving performance
2. Measurement Specialties, Inc. Announces Impact of German Business Tax Reform 2008 Resulting in Lower Combined German Tax Rate - Impacts 2Q08 Reported Financial Results
3. Bee species outnumber mammals and birds combined
4. Sociological research shows combined impact of genetics, social factors on delinquency
5. A combined tooth-venom arsenal revealed as key to Komodo dragons hunting strategy
6. A new radiation therapy treatment developed for head and neck cancer patients
7. St. Jude finds factors that accelerate resistance to targeted therapy in lymphoblastic leukemia
8. UC health news: molecular pathway may predict chemotherapy effectiveness
9. MIT works toward safer gene therapy
10. Intravenous gene therapy protects normal tissue of mice during whole-body radiation
11. Gene, stem cell therapy only needs to be 50 percent effective to create a healthy heart
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Combined stem cell-gene therapy approach cures human genetic disease in vitro
(Date:8/12/2015)... MINNETONKA, Minn. , Aug. 12, 2015   ... that supports the entire spectrum of clinical research, is ... ™ , the company,s comprehensive SaaS-based eClinical technology platform, ... growth during the first two quarters of 2015.   ... and second largest quarters measured by contract value sold ...
(Date:8/10/2015)... and TELTOW, Germany , August ... (SMI), a world leader in Eye Tracking Technology ... of its OEM Eye Tracking Platform for integration into ... Platform contains reference designs for seamless integration of eye ... virtual reality HMDs and augmented reality smart glasses. Omnivision,s ...
(Date:8/5/2015)... 2015 According to a new ... Software, Services, Vertical (BFSI, Airport, IT & Telecom, Utilities, ... Forecast to 2020", published by MarketsandMarkets, defines and segments ... various segments. The global PIAM Market is estimated to ... by 2019, at a CAGR of 14.9 % from ...
Breaking Biology News(10 mins):MedNet Solutions Experiences Explosive Corporate Growth 2SMI Completes OEM Eye Tracking Platform to Cover All Consumer Displays 2SMI Completes OEM Eye Tracking Platform to Cover All Consumer Displays 3Physical Identity and Access Management Market Worth $546.2 Million by 2019 2Physical Identity and Access Management Market Worth $546.2 Million by 2019 3Physical Identity and Access Management Market Worth $546.2 Million by 2019 4
... team at Stevens Institute of Technology is working to ... thoracic surgeries. Five undergraduate Biomedical Engineering students have invented ... catheter design and introduces a potentially profitable new product ... of the Stevens Innovative Fluid Extraction System (SIFES) Senior ...
... Asia for its medicinal benefits has been found to ... development in mice during early trials, new Queensland University ... (PSP), which is extracted from the ,turkey tail, mushroom, ... suppress tumour formation in mice, an article written by ...
... Boston, MA The physical forces that guide how cells ... a coordinated fashion inside the living body are poorly understood. ... the Institute for Bioengineering of Catalonia (IBEC) have, for the ... collective cellular migration. Their surprising conclusion is that the cells ...
Cached Biology News:Stevens thoracic catheter senior design team takes 1st place at regional ISPE competition 2Stevens thoracic catheter senior design team takes 1st place at regional ISPE competition 3Mushroom compound suppresses prostate tumors 2The dance of the cells: A minuet or a mosh? 2The dance of the cells: A minuet or a mosh? 3
(Date:8/28/2015)... -- According to a new market research ... Thawing Equipment, Alarms), by Biospecimen (Human Tissue, Stem Cells, ... - Global Forecast to 2020", published by MarketsandMarkets, the ... Million by 2020 from USD 2,150.48 Million in 2015, ... 2020. Browse 74 Tables and 95 ...
(Date:8/28/2015)... ... ... of FireflySci Inc. (FFS) in early 2015, the company continues to shake up the cuvette ... in a lab coat and Large Marge have been posted on the company’s Facebook page ... is proud to be the only cuvette manufacturer is the world that has an online ...
(Date:8/27/2015)... -- The National Necrotizing Fasciitis Foundation (NNFF) ... doctors at Vanderbilt University Hospital in Nashville, ... Dr. John Crew , director of the ... in Daly City, CA. ... by Dr. Crew, that had previously saved the lives ...
(Date:8/27/2015)... ... 27, 2015 , ... Inc. Magazine released its annual list of the 5,000 ... in growth for the three years through 2014. Being named to the Inc. 5000 ... in the country. , “We are thrilled to make the Inc. 5000 list ...
Breaking Biology Technology:Biopreservation Market Worth 3,731.03 Million USD by 2020 2Biopreservation Market Worth 3,731.03 Million USD by 2020 3Biopreservation Market Worth 3,731.03 Million USD by 2020 4Biopreservation Market Worth 3,731.03 Million USD by 2020 5Cuvette Manufacturer FireflySci Launches Back to School Deals with Complimentary Shipping On All Orders 2Patient's Life and Eyes Saved from Gruesome Flesh-Eating Infection Consuming Her Face 2Patient's Life and Eyes Saved from Gruesome Flesh-Eating Infection Consuming Her Face 3iLab Solutions Named to 2015 Inc. 5000 List 2iLab Solutions Named to 2015 Inc. 5000 List 3
... eye uses tiny muscles to focus on different distances. ... some sensors do not require these external controls. , ... Engineering are investigating a liquid lens design that ... in a variety of commercial applications, including medical diagnostic ...
... Wis. - Although the third major storm ... Chris, fizzled into a low-pressure system recently, apprehensions ... circulate across the country. , ,A Wisconsin telecommunications ... that apprehension away. , ,To address concerns of ...
... Inc. , a supplier of DNA microarrays for genome ... experience managing large entrepreneurial growth companies. , ,David Snyder ... raising, finance, and general management in a range of ... ,In a statement released by NimbleGen, President and CEO ...
Cached Biology Technology:"Smart" lenses hold commercial potential 2Wisconsin firm leads testing of alerts by cell phone 2Wisconsin firm leads testing of alerts by cell phone 3NimbleGen names CFO with capital-raising past 2
Anti-human C5/C5b, Clone 568, Monoclonal Antibody...
Proline-serine-threonine phosphatase-interacting protein 2...
Rabbit Anti-melatonin (different linkage) Polyclonal Antibody...
Anti-ACTH Immunogen: Porcine ACTH conjugated to BSA. Available Date: 38566...
Biology Products: