Navigation Links
Gene Therapy Shows Promise Against Hereditary Lung Disease

An experimental gene therapy to combat alpha-1 antitrypsin deficiency, a common hereditary disorder that causes lung and liver disease, has caused no harmful effects in// patients and shows signs of being effective, University of Florida researchers say.

In a clinical trial, researchers evaluated the safety of using a so-called gene vector - in this case an adeno-associated virus - to deliver a corrective gene to 12 patients who are unable to produce a protein essential for health called alpha-1 antitrypsin.

“The primary end point in the trial was to see whether it was safe to give patients this gene transfer vector and then to try to begin to see if we could get the dose into a range where we would begin to replace the missing protein in the blood,” said Terence Flotte, M.D., a pediatrician, geneticist and microbiologist with UF’s College of Medicine and a member of the Powell Gene Therapy Center and the UF Genetics Institute. “We found that we can use this agent safely and we also saw evidence in the patients’ blood that the higher doses successfully introduced the vector DNA. In one patient we saw evidence for a very brief period that some of the alpha-1 protein was being produced, but not at a high enough level to be beneficial.”

The findings appear online today (Nov. 21) in the journal Human Gene Therapy.

Physicians injected doses of the virus containing copies of the gene for alpha-1 antitrypsin into the patients’ upper arms. Essentially, the virus is intended to “infect” patients’ cells with replacement genes that will do the necessary work to produce alpha-1 protein. UF scientists have successfully developed the technique in animal models.

The next step is to test the therapy with a different version of the adeno-associated virus; about 200 variations of the virus exist in nature.

“We have another version of the virus that appears in animal studies to be close to a thousandfold more pot
'"/>




Page: 1 2 3

Related medicine news :

1. Cancer Doctors Okays Controversial Prostate Therapy
2. Consensus on "Combination Therapy" for Breast Cancer
3. Gene Therapy shows promise in treating Hemophilia
4. "Make AIDS Therapy affordable" - Physicians demand
5. Hormone replacement Therapy a headache
6. Simple Therapy
7. Therapy for stopping the spread of cancer cells
8. Gene Therapy Destroys Pancreatic Cancer Cells
9. Letrozole Beats Tamoxifen in Breast Cancer Therapy
10. Garlic Supplements Impede HIV Therapy
11. Gene Therapy For Cystic Fibrosis
Post Your Comments:
*Name:
*Comment:
*Email:
TAG: Gene Therapy Shows Promise Against Hereditary Lung Disease

(Date:9/19/2014)... made from the wonder material graphene has been used ... developing cancer. , The biosensor has been shown ... bioassay tests currently in use, and was able to ... the possibility of a rapid, point-of-care diagnostic tool for ... September, in IOP Publishing,s journal 2D Materials . ...
(Date:9/19/2014)... 2014 Follow us on LinkedIn ... medical imaging market, are engineered to enhance the anatomic ... past three decades have expanded the application roles of ... based agents used for over half a century to ... agents have witnessed sea changes, revolutionizing in the process ...
(Date:9/19/2014)... 2014 The Nordic ... style of eating, is making its way ... Doc’s medical weight loss programs, ... good food which leads to improved health. ... patients to understand how diet can impact ...
(Date:9/19/2014)... Recent financial data reveled ... their sales by 7% in the last quarter of ... can be used as a bellwether to determine how ... data has been proven to show trends with shipments ... Other non-trucking data is largely influenced by international and ...
(Date:9/19/2014)... Baltimore, MD & Salt Lake City, UT (PRWEB) September ... pleased to launch the new Activz Greens Smoothie ... Natural Products Expo East, September 18th-20th in Baltimore, ... powder with 8 organic super foods combined in one ... at Natural Products Expo East, September 18th-20th in Baltimore, ...
Breaking Medicine News(10 mins):Health News:Graphene sensor tracks down cancer biomarkers 2Health News:New Clinical Applications and Advanced Imaging Capabilities Drive Growth in the Imaging Agents Market, According to New Report by Global Industry Analysts, Inc. 2Health News:New Clinical Applications and Advanced Imaging Capabilities Drive Growth in the Imaging Agents Market, According to New Report by Global Industry Analysts, Inc. 3Health News:New Clinical Applications and Advanced Imaging Capabilities Drive Growth in the Imaging Agents Market, According to New Report by Global Industry Analysts, Inc. 4Health News:Diet Doc’s Medical Weight Loss Programs Announce Diet Plans Based on the Same Principles as the New Nordic Diet 2Health News:Diet Doc’s Medical Weight Loss Programs Announce Diet Plans Based on the Same Principles as the New Nordic Diet 3Health News:Diet Doc’s Medical Weight Loss Programs Announce Diet Plans Based on the Same Principles as the New Nordic Diet 4Health News:LeaseQ Announces Good News For The Truck Leasing Industry As Truck Rentals In The U.S Continues to Gain Traction 2Health News:LeaseQ Announces Good News For The Truck Leasing Industry As Truck Rentals In The U.S Continues to Gain Traction 3Health News:New Activz Greens Smoothie with 8 Organic Superfoods & More Launched at Natural Products Expo East 2Health News:New Activz Greens Smoothie with 8 Organic Superfoods & More Launched at Natural Products Expo East 3
... Jeffrey M. Bethony, Ph.D., associate professor of Microbiology, ... professor of Microbiology, Immunology, and Tropical Medicine at ... with colleagues from Khon Kaen University in Thailand ... an sensitive and specific biomarker for the detection ...
... being able to mathematically describe the process by which ... its every function, and dysfunction. Such capability holds enormous ... when and where things go developmentally awry and ... of breakthrough is one of the long-term goals of ...
... 29 (HealthDay News) -- Preventive antiretroviral treatment appears to ... against HIV infection, a new study suggests. HIV, ... unprotected sex and contaminated needles. Immediate treatment after ... previous research has found. More recently, several large randomized, ...
... Adrian Mulholland and Dr Christopher Woods from Bristol,s School ... processing units (GPUs) to simulate the molecular processes that ... the H1N1-2009 strain of influenza commonly known as ... in Biochemistry , provide new insight that could ...
... disorders have uncovered insights into those diseases in ... Focusing on the cohesin complex, a group of ... scientists have discovered mutations that disrupt cohesin, causing ... "We are learning more about how these ...
... This release is available in German . ... to establish 20 Collaborative Research Centres (CRCs) by 1 July ... its spring session in Bonn. The new CRCs will receive ... allowance for indirect project costs) for the initial 4-year funding ...
Cached Medicine News:Health News:GW researchers discover biomarker for advanced bile duct fibrosis and bile duct cancer 2Health News:UC Santa Barbara's Kavli Institute receives 2 grants to explore interface of physics and biology 2Health News:UC Santa Barbara's Kavli Institute receives 2 grants to explore interface of physics and biology 3Health News:Taking Anti-HIV Meds Prior to Exposure May Help Prevent Infection 2Health News:New study shows why swine flu virus develops drug resistance 2Health News:Mutations impair childhood growth and development by disrupting organization of chromosome pairs 2Health News:DFG establishes 20 new Collaborative Research Centres 2Health News:DFG establishes 20 new Collaborative Research Centres 3Health News:DFG establishes 20 new Collaborative Research Centres 4Health News:DFG establishes 20 new Collaborative Research Centres 5Health News:DFG establishes 20 new Collaborative Research Centres 6
(Date:9/19/2014)... -- The American Association for Homecare (AAHomecare) is pleased ... joined the AAHomecare Corporate Partner Program. ... been actively involved in AAHomecare for much of the ... into a major global manufacturer of home medical equipment. ... increase efforts on behalf of the HME sector in ...
(Date:9/19/2014)... DALLAS , September 19, 2014 ... Market by Equipment Type (Benchtop, Mobile, Industrial, & ... Others), Technology (Tray-style, Manifold, & Rotary) & Geography ... by MarketsandMarkets, defines and segments the lyophilization market ... regions with analyses and projections of the market ...
(Date:9/19/2014)... and ORLANDO, Fla. , Sept. 19, ... therapy continues to grow, with the publication of two ... in Otolaryngology – Head and Neck Surgery ... worsening of both objective and subjective measures of sleep ... Inspire therapy for the reduction in obstructive sleep apnea ...
Breaking Medicine Technology:Drive Medical Design & Manufacturing Joins AAHomecare Corporate Partner Program 2Lyophilization Market Worth $5,121.5 Million by 2019 2Lyophilization Market Worth $5,121.5 Million by 2019 3New Data shows Reductions in obstructive sleep apnea (OSA) severity and improvement of quality of life with Inspire therapy 2New Data shows Reductions in obstructive sleep apnea (OSA) severity and improvement of quality of life with Inspire therapy 3New Data shows Reductions in obstructive sleep apnea (OSA) severity and improvement of quality of life with Inspire therapy 4
... CHICAGO, March 7, 2012  UHC, an alliance of the ... of a mobile application for Rx LINK ™ , ... insight into their pharmacy price performance. The mobile app ... can be accessed on an Apple iPad. ...
... Drug Discovery Foundation (ADDF) and the Association for ... their fifth annual research award, Frontotemporal Degeneration ... of the program is to accelerate the development ... The ADDF/AFTD program awarded $300,000 to ...
Cached Medicine Technology:UHC Launches RxLINK(TM) Mobile App to Deliver Instant Data Access for Strategic Pharmacy Purchasing 2UHC Launches RxLINK(TM) Mobile App to Deliver Instant Data Access for Strategic Pharmacy Purchasing 3Awards for Drug Discovery Research for Frontotemporal Degeneration Announced 2Awards for Drug Discovery Research for Frontotemporal Degeneration Announced 3
Used for placement of choledochoscopes, cholangiography catheters and other instruments. Supplied sterile in peel-open packages. Intended for one-time use....
Used for common bile duct exploration. Supplied sterile in peel-open packages. Intended for one-time use....
... Marker directly to the biopsy site helps ... performed. ,The MICROMARK® II Tissue Marker is ... for use with the MAMMOTOME Biopsy System. ... site after a biopsy with the MAMMOTOME ...
... A biopsy with the ... an effective, minimally invasive procedure ... patient management.,Using computer-generated images to ... more accurately pinpoint and map ...
Medicine Products: