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:5/4/2016)... , ... May 04, 2016 , ... ... Continence Nurses (WOCN) Society™ and Canadian Association for Enterostomal Therapy (CAET) will ... time saving and planning tools to attendees and exhibitors for the 2016 WOCN ...
(Date:5/4/2016)... Charles, LA (PRWEB) , ... May 04, 2016 , ... ... Charles, LA area has teamed up with Big Brothers Big Sisters of Southwest Louisiana ... now being accepted here . , Big Brothers Big Sisters of Southwest Louisiana ...
(Date:5/4/2016)... ... May 04, 2016 , ... Washington Wellness Center today announced its tenth anniversary ... What started out as an idea to provide a holistic approach to wellness ... , Developed by Dr. David Swanekamp, Chiropractic Physician , the wellness center offers ...
(Date:5/4/2016)... , ... May 04, 2016 , ... ... 2016. SS&A teamed up with one of the top website design companies to ... contains informative legal articles related to the law firm's main practice areas. These ...
(Date:5/4/2016)... ... May 04, 2016 , ... Momsense, ... nursing mothers. The company’s patented technology, The Smart Breastfeeding Meter, is designed to ... that the technology is now available for purchase at Target.com . ...
Breaking Medicine News(10 mins):
(Date:5/4/2016)... YORK , May 4, 2016 ... Share, Development, Growth and Demand Forecast to 2022 - Industry ... User (Hospital, Diagnostic Center and Others)" by P&S Market Research, ... $4,894.3 million in 2015, and it is expected to grow ... type, the high slice type segment is expected to witness ...
(Date:5/4/2016)... 4, 2016 In March, ... series of free workshops across Africa ... requirements for Good Distribution Practices (GDP). Good Distribution ... that products are consistently stored, transported and handled under ... or product specification. Only a few years ago, there ...
(Date:5/4/2016)... , May 4, 2016 ... addition of the  "Global Multiple Myeloma Market ... to their offering.       (Logo: ... Myeloma Market and Competitive Landscape Highlights 2016, ... products, Multiple Myeloma epidemiology, Multiple Myeloma market ...
Breaking Medicine Technology: