Navigation Links
Gene therapy for hereditary lung disease advances

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 potent at making pro
'"/>

Source:University of Florida


Page: 1 2 3

Related biology news :

1. Adding Radiation Therapy To Chemotherapy Improves Survival In Patients With High-risk Breast Cancer
2. Columbia research lifts major hurdle to gene therapy for cancer
3. Combination therapy boosts effectiveness of telomere-directed cancer cell death
4. Gene therapy converts dead bone graft to new, living tissue
5. Study identifies predictors of HIV drug resistance in patients beginning triple therapy
6. New imaging method gives early indication if brain cancer therapy is effective, U-M study shows
7. Muscle-targeted gene therapy reverses rare muscular dystrophy in mice
8. New therapy for HIV/AIDS eliminates needles and excessive toxicity
9. New Treatment Rivals Chemotherapy For Lymphoma, Study Finds
10. Gene therapy for Parkinsons disease moves forward in animals
11. Pulsating ultrasound enhances gene therapy for tumors
Post Your Comments:
*Name:
*Comment:
*Email:
TAG: Gene therapy for hereditary lung disease advances

(Date:5/22/2015)... May 22, 2015  NXT-ID, Inc. (NASDAQ: NXTD ) ... will be ending its promotional pricing and making the Wocket ... Laurence Savin , Head of Marketing said, "We,ve extended ... until the end of May. To get your wocket at ... we open the store on June 1, you,ll receive a ...
(Date:5/21/2015)... The Sync Project™ , a global collaboration harnessing ... a collaborative partnership with Berklee,s Institute for Creative ... on original research, joint course development and an internship ... Sync Project and BerkleeICE are exploring the launch of ... academic year.  The course will focus ...
(Date:5/19/2015)... Sweden , May 18, 2015 ... fingerprint sensors FPC1020, FPC1025, FPC1145, FPC1155 and FPC1035 from one ... from June until and including Q3 2015 and the sensors ... . Communicated order values for 2015 hereby amount to ... for Q1 2015 of 140 MSEK and a number of ...
Breaking Biology News(10 mins):NXT-ID, Inc Announces Retail Price For Wocket Smart Wallet And Extends Discounted Promotional Price Till End Of May 2NXT-ID, Inc Announces Retail Price For Wocket Smart Wallet And Extends Discounted Promotional Price Till End Of May 3The Sync Project Announces Partnership with Berklee College of Music's Institute for Creative Entrepreneurship 2The Sync Project Announces Partnership with Berklee College of Music's Institute for Creative Entrepreneurship 3Fingerprint Cards Receives Touch Fingerprint Sensor Order of SEK 230 Million and Raises Revenue Guidance for 2015 2
... beat about the same number of times in its lifetime ... while an elephant sees 70 winters come and go? Why ... Why has nature chosen such radically different forms as the ... of a tiger? These questions have puzzled life ...
... and elevated levels of the ,stress hormone, cortisol are up ... those who show neither trait, according to research funded by ... in the Proceedings of the National Academy of Sciences ... first biomarker a biological signpost for major, or ...
... endemic infectious diseases of humans have been largely contained, ... animal health. Such emerging infectious diseases are not ... wildlife ecosystems; the finely-tuned dynamic balance of which is ... scale and manner of livestock production and marketing, the ...
Cached Biology News:How evolution shapes the geometries of life 2How evolution shapes the geometries of life 3How evolution shapes the geometries of life 4First biological marker for major depression could enable better diagnosis and treatment 2First biological marker for major depression could enable better diagnosis and treatment 3First biological marker for major depression could enable better diagnosis and treatment 4Zoonotic diseases and global viral pandemics 2Zoonotic diseases and global viral pandemics 3
(Date:5/29/2015)... Market Publishers Ltd and Shanghai Chem-Report ... agreement for quality market research promotion on Internet. ... research reports worked out by Chem-Report. , Natalie ... the agreement: “Chem-Report is a Chinese company involved ... markets on global and country levels. The firm ...
(Date:5/29/2015)... 29, 2015  Caris Life Sciences ® , ... promise of precision medicine, today announced the presentation ... clinical utility of Caris Molecular Intelligence ® , ... helping to identify targeted treatment options for patients ... the studies, results of which are to be ...
(Date:5/29/2015)... Seoul Semiconductor (CEO: Chung-hoon Lee, http://www.seoulsemicon.com ... 28th announced that it will increase the global ... efficient and high reliable LED product line-up for ... has developed efficient and reliable LED packages for ... for headlamps to global auto makers. Seoul Semiconductor’s ...
(Date:5/28/2015)... 2015  Quest Diagnostics Incorporated (NYSE: DGX ), ... that it is scheduled to speak at the Jefferies ... Rusckowski , the company,s President and CEO, will discuss ... presentation is scheduled for Tuesday, June 2, 2015 at ... be webcast live during the conference and will be ...
Breaking Biology Technology:Market Publishers Ltd and Chem-Report Sign Partnership Agreement 2Market Publishers Ltd and Chem-Report Sign Partnership Agreement 3Multi-Platform Tumor Profiling from Caris Life Sciences Identifies Targeted Treatment Options for Patients with Rare and/or Aggressive Tumors 2Multi-Platform Tumor Profiling from Caris Life Sciences Identifies Targeted Treatment Options for Patients with Rare and/or Aggressive Tumors 3Multi-Platform Tumor Profiling from Caris Life Sciences Identifies Targeted Treatment Options for Patients with Rare and/or Aggressive Tumors 4Multi-Platform Tumor Profiling from Caris Life Sciences Identifies Targeted Treatment Options for Patients with Rare and/or Aggressive Tumors 5Multi-Platform Tumor Profiling from Caris Life Sciences Identifies Targeted Treatment Options for Patients with Rare and/or Aggressive Tumors 6Seoul Semiconductor Increases the Global Automotive LED Market Share with its High Efficient and Reliable LEDs for Automotive Lightings 2Quest Diagnostics to Speak at the Jefferies 2015 Global Healthcare Conference 2
... Dynamics and GridGain teamed up to develop the largest publicly known ... ... Francisco, CA (PRWEB) August 4, 2008 -- Following up on their ... the largest publicly known grid scalability test for Amazon EC2. The ...
... SUNRISE, Fla., Aug. 1 Bioheart, Inc. (Nasdaq:,BHRT) ... of,Bioheart, will present a corporate overview Thursday, August 21, ... Conference at the Marriott,Marquis in New York. A live ... internet by visiting and registering to attend at:, http://www.redchip.com ...
... On the 25th/26th of July the first,ever transplantation of ... of the Technical University of Munich. The responsibility for ... Hand Surgery. The,operation, performed by a team of approx. ... physician for Plastic Surgery and Hand Surgery),and Prof. Edgar ...
Cached Biology Technology:Grid Dynamics Demonstrates Linear Scalability up to 512 Nodes with GridGain Software on Amazon EC2 Cloud 2Grid Dynamics Demonstrates Linear Scalability up to 512 Nodes with GridGain Software on Amazon EC2 Cloud 3Bioheart, Inc. to Present at Red Chip Investor Conference in New York 2The First Ever Transplantation World-Wide of Entire Arms at the "Klinikum rechts der Isar" in Munich 2
... Dyad Disciple thermal cycler is a dual-bay ... also be operated independently, using a PC. ... interchangeable Alpha units (sample-holder, heat-pump assemblies), which ... the Chromo4 real-time detector. Peltier heat pumps ...
...
I-Ab MHC Class II...
MHC II (HLA-DP)...
Biology Products: