Navigation Links
Carnegie Mellon researchers to develop new drug delivery system
Date:11/2/2007

PITTSBURGH Carnegie Mellon Universitys Stefan F. Zappe is using adult neural stem cells to develop a new stem cell-based drug delivery therapy that may ultimately help treat a variety of inherited genetic disorders like Hunter syndrome.

Zappe, an assistant professor of biomedical engineering at Carnegie Mellon, and his graduate student Sasha Bakhru, are creating genetically engineered adult neural stem cells for delivery to patients brains, where they will be programmed to produce an essential missing protein. In Hunter syndrome, for example, patients are lacking the enzyme iduronate-2-sulfatase that helps cells break down certain waste products. One in every 130,000 boys is born with the rare but deadly genetic disorder.

Zappe, who is working with Dr. Raymond Sekula, a neurosurgeon at Allegheny General Hospital, said he selected adult neural stem cells for his work because they can be harvested from a patients brain, have the potential to be multiplied outside of the body, can be genetically engineered, can disperse within the brain once re-implanted and can replace all major cell types of the brain.

To support their therapeutic goals, Zappe and his team have developed cell-instructive microcapsules that contain neural stem cells. These microcapsules efficiently control whether stem cells proliferate (multiply), differentiate into more specialized cell types like neurons and to what extent implanted stem cells will be allowed to migrate to the host tissue.

Zappe will be using these caviar-sized capsules specifically for rapid manipulation of stem cells outside the body and for reliable delivery of stem cells to the brain. The acute inflammatory response that usually occurs from implantation would normally cause implanted neural stem cells to differentiate into mature cell types that are not able to migrate extensively. Encapsulated stem cells will be protected from such premature differentiation.

Once the brain has healed from the initial implant of the encapsulated stem cells, the stem cells are genetically engineered to produce an enzyme that eats the microcapsule, freeing the neural stem cells. The stem cells can then migrate deep into the surrounding brain tissue where they provide the missing enzyme.

We are particularly interested in targeting the brain because this area of the body is protected by the so-called blood-brain barrier that has been very difficult to penetrate with therapeutic enzymes that are usually injected into the patients bloodstream, Zappe said. Zappe and Sekula are working to develop technologies that will ultimately enable clinicians to harvest neural stem cells from a patient, genetically engineer them from outside the body and then re-implant them and remotely control their actions in non-invasive ways.

By using inducible gene expression, we hope to provide physicians with external control over capsule degradation and the amount of therapeutic enzyme released into the brain by engineered cells as determined by the dose of drugs that are given to the patient in pill form, Zappe said.

Hunter syndrome is a devastating illness affecting more than 500 children in the U.S. alone. Over time, toxic waste products accumulate in the cells of the body, and, although progression of the disease varies, the majority of children die in their teens. If we can reliably provide the missing enzyme iduronate-2-sulfatase to the central nervous system of these children, we may change the course of this disease. Our technology and methodology also will likely have far-reaching implications for hundreds of other diseases of the central nervous system, Sekula said.


'/>"/>

Contact: Chriss Swaney
swaney@andrew.cmu.edu
412-268-5776
Carnegie Mellon University
Source:Eurekalert

Related biology news :

1. Carnegie Mellon scientists develop tool that uses MRI to visualize gene expression in living animals
2. Robot-based system developed at Carnegie Mellon detects life in Chiles Atacama desert
3. Green catalyst destroys pesticides and munitions toxins, finds Carnegie Mellon University
4. Carnegie Mellon University research reveals how cells process large genes
5. Carnegie Mellon cyLab researchers work to develop new red tide monitoring
6. Team led by Carnegie Mellon University scientist finds first evidence of a living memory trace
7. Carnegie Mellon scientists create PNA molecule with potential to build nanodevices
8. Carnegie Mellon U. transforms DNA microarrays with standard Internet communications tool
9. Carnegie Mellon develops non-invasive technique to detect transplant rejection at cellular level
10. Carnegie Mellon scientists show brain uses optimal code for sound
11. DNA conclusive yet still controversial, Carnegie Mellon professor says
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/24/2016)... -- Cercacor today introduced Ember TM Sport Premium ... measure hemoglobin, Oxygen Content, Oxygen Saturation, Perfusion Index, ... approximately 30 seconds. Smaller than a smartphone, using only ... key data about their bodies to help monitor these ... Hemoglobin carries oxygen to muscles. When hemoglobin and ...
(Date:11/19/2016)... Securus Technologies, a leading provider of civil ... corrections and monitoring, announced today that it has offered ... an independent technology judge determine who has the largest ... telephone calling platform, and the best customer service. ... of what we do – which clearly is not ...
(Date:11/15/2016)... ROCKVILLE, Md. , Nov. 15, 2016 /PRNewswire/ ... clinical company developing therapeutics focused on the gut ... public offering of 25,000,000 shares of its common ... its common stock at a price to the ... gross proceeds to Synthetic Biologics from the offering, ...
Breaking Biology News(10 mins):
(Date:12/2/2016)... (PRWEB) , ... December 02, ... ... (ETC), a consortium of pharmaceutical and biotechnology companies dedicated to collaboratively developing ... companies interested in supplying a vendor-supported, portable online UHPLC, with robust, probe-based ...
(Date:12/2/2016)... , December 2, 2016 The ... 2021, growing at a CAGR of 7.3% during the forecast period ... hospitals and diagnostic laboratories segment accounted for the largest share of ... ... report on global immunohistochemistry (IHC) market spread across 225 pages, profiling ...
(Date:11/30/2016)... 30, 2016  GenomOncology today announced the appointment of ... Affairs.  Dr. Coleman will oversee clinical content ... knowledge-enabled platform. The GenomOncology software suite empowers molecular pathologists with ... and clinical decision support, from quality control through reporting. ... , , ...
(Date:11/30/2016)... 2016 Biotest Pharmaceuticals Corporation (BPC), a leading ... the addition of its newest plasma collection center located ... . The 15,200 square foot state-of-the-art facility officially opened ... brings the total number of BPC,s plasma collection centers ... BPC,s Chief Executive Officer said "We are pleased to ...
Breaking Biology Technology: