Navigation Links
Gene therapy research from Penn Vet & Scheie Eye Inst. cures retinitis pigmentosa in dogs
Date:1/23/2012

r reversing XLRP but potentially for any form of photoreceptor degeneration," Aguirre said. "Altered inner retinal wiring is a common feature for these diseases that has been considered irreversible.

"The study required a combination of genetic tools and surgical technique to make sure the therapy targeted only the diseased cells. The viral vector had to be injected in the sub-retinal space so as to be in close proximity to the photoreceptors. Likewise, you need to ultimately deliver the therapy to the right location of the retina," Aguirre said.

"In the human disease, careful characterization of the areas of the retina that need to be treated is going to be critical for therapy to succeed in the clinic," Cideciyan said.

The genetic aspect of the viral vector used in this study involved a double safeguard. The first safety feature was to use a viral vector that is known to predominantly target both rods and cones but not other cells. The second safeguard involved attaching the healthy RPGR gene to a "promoter," a piece of genetic code that would "switch on" the gene only if the virus penetrated the correct cell.

Selecting the right promoter was critical; the lead researchers at the University of Florida, William W. Hauswirth and Alfred S. Lewin, had to find one that that would be turned on exclusively in rods and cones. This way, even if the virus made its way to a non-photoreceptor cell, that cell would not start activating the RPGR gene.

That both the promoter and the RPGR gene it activates are taken from humans is a strong sign that the treatment may be translatable to patients.

"While there is still much work to do to assess long-term efficiency and safety with this approach, there is hope that this vector and knowledge could be used in a few years to treat the many patients losing vision from XLRP," Jacobson said.
'/>"/>

Contact: Evan Lerner
elerner@upenn.edu
215-573-6604
University of Pennsylvania
Source:Eurekalert

Page: 1 2 3

Related biology technology :

1. Lixte Biotechnology Holdings Announces Filing of a New Patent Application for Cancer Therapy Based on Targeting a Biomarker by Novel Investigational Agents
2. Reliant Technologies Announces Canadian Launch of Fraxel re:pair(R) Laser: A New Class of Treatment Therapy for CO2 Laser Skin Resurfacing
3. FDA Approves ALOXI(R) (Palonosetron HCl) Capsules for Prevention of Acute Chemotherapy-induced Nausea and Vomiting
4. Regenerative Medicine Appoints Cell Therapy Group Consultant to Editorial Board
5. Gleevec Receives FDA Priority Review as First Therapy to Reduce Recurrence of Gastrointestinal Stromal Tumors After Surgery
6. Brain Cancer Patients at Two Leading Hospitals Treated Using Fast and Efficient RapidArc Radiotherapy Technology from Varian Medical Systems
7. Progenitor Cell Therapy Secures Industry Accreditation
8. Nucletron Announces Market Release of First Commercially Available Software to Review and Share Dicom-Based Treatment Plans for Radiotherapy
9. Doctors in the US and Netherlands Using Fast RapidArc Radiotherapy Technology to Help Fight Cancer are Now Treating Tumors of the Head and Neck
10. First Coast Oncology to Offer Next Generation Proton Therapy
11. Varian to Showcase RapidArc(TM) Radiotherapy Technology Enhancements at ASTRO 2008
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/25/2014)... The report “Service Quality Management and Telco ... Type - Global Advancements, Worldwide Forecasts & Analysis (2014-2019)” ... and forecasting of revenues. , Browse 152 market ... in-depth TOC on “Service Quality Management (SQM) and Telco ...
(Date:12/24/2014)... DALLAS and NEW YORK , ... a biopharmaceutical company advancing patient care in critical areas, announced ... of common stock, and warrants to purchase up to an ... of $4.00 per share and $.01 per warrant.  The warrants ...
(Date:12/24/2014)... BOTHELL, Wash. , Dec. 24, 2014   BioLife Solutions ... manufacturer and marketer of proprietary clinical grade hypothermic storage ... thermal shipping products for cells and tissues  ("BioLife" or ... Annual Meeting of Stockholders on May 4, 2015 (the "Annual ...
(Date:12/24/2014)... 23, 2014 GMO corn cases filed across ... in the process of being consolidated in a Kansas federal ... Syngenta AG MIR 162 Corn Litigation, MDL No. 2591 in ... Management of the Syngenta GMO corn multidistrict litigation (MDL) has ...
Breaking Biology Technology:Service Quality Management Market worth $2.1 Billion & Telco Customer Experience Management Market worth $2.3 Billion by 2019 – New Report by MarketsandMarkets 2Service Quality Management Market worth $2.1 Billion & Telco Customer Experience Management Market worth $2.3 Billion by 2019 – New Report by MarketsandMarkets 3Service Quality Management Market worth $2.1 Billion & Telco Customer Experience Management Market worth $2.3 Billion by 2019 – New Report by MarketsandMarkets 4PlasmaTech Biopharmaceuticals, Inc. Announces Closing of Public Offering 2PlasmaTech Biopharmaceuticals, Inc. Announces Closing of Public Offering 3BioLife Solutions Sets Date for Annual Meeting of Stockholders 2Carey Danis & Lowe Reports on Syngenta GMO Corn Transfer Order 2
... Wis. - As law professors at major universities, Rebecca ... case perfectly clear: they are not calling for repeal of ... patent their inventions. They are advocating an adjustment that ... University of Michigan and Rai of Duke University ...
... - An agreement struck to accelerate the transformation of ... visions of a medical discovery triangle connecting Milwaukee, Madison, ... milestone in the drive to facilitate scientific collaboration and ... formally joined forces with WiSys Technology Foundation, Inc. ...
... Madison, Wis. - Quick quiz: Which Wisconsin institutions spend ... , ,Naming No. 1 is relatively easy: the University ... academic R&D centers in the United States. Second on the ... million), which ranks among the nation's top 100 medical research ...
Cached Biology Technology:Professors want to tweak, not overhaul, patent law 2Professors want to tweak, not overhaul, patent law 3WiSys to patent Marshfield Clinic discoveries 2Marshfield Clinic and UW combine to speed tech growth 2Marshfield Clinic and UW combine to speed tech growth 3Marshfield Clinic and UW combine to speed tech growth 4
(Date:1/22/2015)... ("Company") (BM&FBOVESPA: VLID3 ON), is pleased to inform its shareholders ... Washington,s Department of Licensing, in the United States, awarded ... for driver,s licenses and identification cards to its subsidiary, Valid ... start in January 2015, with enrollment and card production starting ...
(Date:1/22/2015)... MADISON, Wis. , Jan. 15, 2015  BellBrook ... for drug discovery, announced the launch of a ... of the company,s Transcreener UDP Assay, a high ... enzymes. The new assay will allow for sensitive ...
(Date:1/22/2015)... YORK , Jan. 21, 2015  Analyst Report Issued by ... Card Brands report showed that planet-wide, transactions at merchants on ... while the total number of credit, debit, and prepaid cards ... U.S. were victims of fraudulent card usage in 2012, and ...
Breaking Biology News(10 mins):Valid USA Signs Contract For Washington Driver's License Issuance 2BellBrook Labs Launches a High Throughput Screening Assay for Glycosyltransferases with TR-FRET Readout 2$4.80 Near Term Price Target with "Buy" given by CapRock Research for NXT-ID, Inc. (NASDAQ: NXTD) issued by Small Cap IR 2
... Scientists at Case Western Reserve University School of Medicine ... that grow into the protective myelin coating on nerves in ... treatments for multiple sclerosis, cerebral palsy and other demyelinating diseases ... published in the online issue of Nature Methods , ...
... Ibuprofen, naproxen, and related non-steroidal anti-inflammatory drugs (NSAIDs) ... still have some secrets to reveal about how ... new insights into the actions of NSAIDs. Their ... raise the possibility of developing a new class ...
... Chalmers University of Technology have built a very simple ... directions, even though the antenna is smaller than the ... online journal Nature Communications this week ... very low concentrations of gases or biomolecules. A ...
Cached Biology News:Mice stem cells guided into myelinating cells by the trillions 2Mice stem cells guided into myelinating cells by the trillions 3Study puts a new spin on ibuprofen's actions 2Bimetallic nanoantenna separates colors of light 2
Goat polyclonal to T7 tag (Agarose) Immunogen: Goats were immunized with MASMTGGQQMG (T7) conjugated to KLH.After multiple immunizations in Freund's adjuvant, serum was collected....
Insect Cell Lysis Buffer 50 ml...
Mouse monoclonal [11C9] to Zeralenone ( Abpromise for all tested applications). Antigen: Chemical / Small Molecule conjugated to BSA: Zeralenone...
Fas-ligand rabbit polyclonal antibody...
Biology Products: