HOME >> BIOLOGY >> NEWS
Gene therapy a possibility for metachromatic leukodystrophy?

Metachromatic leukodystrophy (MLD) is an inherited disease that causes progressively more severe neurological defects that result in death early in life. Individuals with MLD have a genetic defect that means they lack a protein known as ARSA. There are currently no therapies for MLD, largely because the barrier between the blood supply and brain -- which tightly regulates the substances that can enter the brain -- provides an obstacle that has yet to be overcome. In a study appearing in the November issue of the Journal of Clinical Investigation, Alessandra Biffi and colleagues from the San Raffaele Scientific Institute in Italy, now show that the neurological defects in mice lacking ARSA can be corrected by treatment with hematopoietic stem progenitor cells (HSPCs) genetically modified to express high levels of ARSA. Importantly, ASRA was detected in the neurons of the treated mice and was derived from microglial cells, which differentiate from HSPCs. The successful correction of the protein defect in neurons and of the neurological defects led the authors to suggest that HSPC gene therapy might be efficacious for the treatment of individuals with MLD.


'"/>

Contact: Karen Honey
press_releases@the-jci.org
212-342-4159
Journal of Clinical Investigation
1-Nov-2006


Page: 1

Related biology news :

1. Testosterone replacement therapy: How safe for aging men?
2. Progesterone therapy and preterm birth: More evidence helps identify women who can benefit
3. Aggressive therapy best for certain AML patients
4. Under magnetic force, nanoparticles may deliver gene therapy
5. 47th Interscience Conference on Antimicrobial Agents and Chemotherapy
6. Stem cell therapy rescues motor neurons in ALS model
7. Study identifies new gene therapy tools for inherited blindness
8. Breast cancer and hormone therapy -- A looking-glass mirror?
9. Radiation therapy combined with microsurgery shows promise for curing injured spinal cord
10. Nodal status is best predictor of outcome after neoadjuvant therapy for esophageal cancer
11. Scientists follow familiar TRAIL to new cancer therapy

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:8/14/2018)... ... 13, 2018 , ... Career fairs provide an ideal setting ... systems at once. However, healthcare professionals often have hectic schedules with little spare ... healthcare providers who wish to explore opportunities and learn about multiple employers from ...
(Date:8/14/2018)... , ... August 13, 2018 , ... ... products, the Staticmaster® P-2021-5502 and P-2021-5602 Cleanroom Ionizing Gun. , The ionizing ... Nitrogen Air Gun, P-2021 ionizing cartridge, 0.5-micron filter and electropolished OSHA-compliant tip. This ...
(Date:8/14/2018)... Fla. (PRWEB) , ... August 14, 2018 , ... ... activity and sleep monitoring solutions for the global scientific community, announced today the ... focus of this new division is to foster and support scientific collaborations with ...
Breaking Biology News(10 mins):
(Date:8/9/2018)... ... August 08, 2018 , ... InTouchMD , the ... announced that they will be exhibiting at Biotech Week Boston , September 4th ... showcasing its Pulse platform, healthcare’s most powerful all in one marketing solution. ...
(Date:8/7/2018)... ... August 06, 2018 , ... Slone Partners, North America’s ... of Dr. Paul Harney as President, North America at Prescient Healthcare Group. Dr. ... client satisfaction. He was also named to Prescient’s Board of Directors. , ...
(Date:7/31/2018)... ... July 31, 2018 , ... ... years, announces the launch of their new website. The new website has been ... the site. Mahoney partnered with Proceed Innovative to build a website created for ...
(Date:7/25/2018)... MATEO, Calif. (PRWEB) , ... July 25, 2018 ... ... for in vivo research utilizing its state-of-the-art Digital Vivarium™ and Vium Cloud approach ... Schaevitz, Ph.D., to Chief Scientific Officer. Additionally, the company announced the appointment of ...
Breaking Biology Technology:
Cached News: