Navigation Links
Researchers discover gene therapy to prevent progression of emphysema
Date:12/21/2009

(Boston) Researchers from Boston University School of Medicine (BUSM) have discovered a new gene therapy that may prevent the progression of emphysema. The study, which appears on-line in the Journal of Clinical Investigation, describes a method to express therapeutic genes in lung tissue for a lifetime after only a single treatment.

Alpha-1 Anti-trypsin Deficiency is the most common inherited form of emphysema seen in young people due to a mutation in the Alpha-1 Anti-trypsin gene. This genetic disease predisposes affected individuals to early emphysema and cirrhosis of the liver.

According to the researchers, gene transfer into specific cell lineages in vivo remains an attractive yet elusive approach for correcting inherited mutations. Although a variety of techniques have been developed to deliver DNA molecules to cells in vitro, in vivo gene transfer has been limited in many cell types by inefficient gene delivery as well as the limited life-span of differentiated cell types

Using mice, the BUSM researchers discovered a system to deliver genes selectively to as many as 70 percent of a mouse lung's alveolar macrophages (AM), a key cell type contributing to emphysema.

"We applied this novel approach to achieve sustained in vivo expression of normal human alpha-1 antitrypsin (hAAT) protein at levels able to ameliorate emphysema in mice," said senior author Darrell Kotton, MD, an associate professor of medicine and pathology and co-director, Center for Regenerative Medicine at BUSM. "The lung macrophages carrying the therapeutic gene survived in the lungs air sacks for the two-year lifetime of the treated mice following a single intra-tracheal injection of the lentiviral vector we had engineered," he added.

Kotton and his colleagues utilized this method of gene transfer to achieve localized secretion of therapeutic levels of human alpha-1 antitrypsin (hAAT) protein in lung epithelial lining fluid. "The progression of emphysema in mice exposed to elastase was significantly improved by the gene therapy as evidenced by improvements in lung compliance and alveolar size," said Andrew Wilson, MD, lead author of the study and an assistant professor of medicine at BUSM.

According to the researchers after 24 weeks of sustained gene expression, no humoral or cellular immune responses to the human hAAT protein were detected. "Our results challenge the dogma that lung macrophages are short-lived and suggest these differentiated cells as a target cell that may be considered for in vivo gene therapy applications including the sustained correction of hAAT deficiency," added Wilson.


'/>"/>

Contact: Gina M. DiGravio
gina.digravio@bmc.org
617-638-8480
Boston University Medical Center
Source:Eurekalert

Related medicine news :

1. Stanford researchers find culprit in aging muscles that heal poorly
2. UCLA researchers identify markers that may predict diabetes in still-healthy people
3. Mayo Clinic researchers discover new diagnostic test for detecting infection in prosthetic joints
4. Bipolar disorder relapses halved by Melbourne researchers
5. Cell that triggers symptoms in allergy attacks can also limit damage, Stanford researchers find
6. High and mighty: first common height gene identified by researchers behind obesity gene finding
7. Researchers estimate about 9 percent of US children age 8 to 15 meet criteria for having ADHD
8. Majority of 2.4 Million U.S. Children With ADHD Not Diagnosed or Consistently Treated, According to New Gold Standard Study by Cincinnati Childrens Researchers
9. Researchers develop long-lasting growth hormone
10. Jefferson immunology researchers halt lethal rabies infection in brain
11. Purdue researchers develop technology to detect cancer by scanning surface veins
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/25/2016)... D.C. (PRWEB) , ... June 25, 2016 , ... ... discuss health policy issues and applications at AcademyHealth’s Annual Research Meeting June 26-28, ... their work on several important health care topics including advance care planning, healthcare ...
(Date:6/25/2016)... FL (PRWEB) , ... June 25, 2016 , ... The temporary closing of Bruton Memorial ... Plant City Observer , brings up a new, often overlooked aspect of head lice: the ... closing for fumigation is not a common occurrence, but a necessary one in the event ...
(Date:6/25/2016)... ... June 25, 2016 , ... First Choice Emergency Room ... Dr. Sesan Ogunleye, as the Medical Director of its new Mesquite-Samuell Farm facility. ... of our new Mesquite location,” said Dr. James M. Muzzarelli, Executive Medical Director of ...
(Date:6/25/2016)... ... June 25, 2016 , ... Dr. ... from injury. Recently, he has implemented orthobiologic procedures as a method for treating ... one of the first doctors to perform the treatment. Orthobiologics are substances that ...
(Date:6/25/2016)... (PRWEB) , ... June 25, 2016 , ... Conventional wisdom ... of success. In terms of the latter, setting the bar too high can result ... more than just slow progress toward their goal. , Research from PsychTests.com ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... June 24, 2016 The Academy of Managed ... recommendations that would allow biopharmaceutical companies to more ... that make formulary and coverage decisions, a move that ... new medicines. The recommendations address restrictions in ... on the drug label, a prohibition that hinders decision ...
(Date:6/24/2016)... India , June 24, 2016 ... Needles Market by Type (Standard Pen Needles, Safety Pen ... Therapy (Insulin, GLP-1, Growth Hormone), Mode of Purchase (Retail, ... by MarketsandMarkets, This report studies the market for the ... expected to reach USD 2.81 Billion by 2021 from ...
(Date:6/23/2016)... , June 23, 2016  MedSource announced ... as its e-clinical software solution of choice.  This ... best possible value to their clients by offering ... The preferred relationship establishes nowEDC as the EDC ... for MedSource,s full-service clients.  "nowEDC has long been ...
Breaking Medicine Technology: