Navigation Links
Gene therapy achieves early success against hereditary bleeding disorder
Date:12/10/2011

(MEMPHIS, Tenn. December 10, 2011) Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K.

The findings of the six-person study mark the first proof that gene therapy can reduce disabling, painful bleeding episodes in patients with the inherited blood disorder. Results of the Phase I study appear in today's online edition of the New England Journal of Medicine. The research is also scheduled to be presented December 11 at the 53rd annual meeting of the American Society of Hematology in San Diego.

Four study participants stopped receiving protein injections to prevent bleeding episodes after undergoing the therapy and have not suffered spontaneous bleeding. Several have also participated in marathons and other activities that would have been difficult prior to gene therapy. The study volunteers were all treated at the Royal Free Hospital in London under the care of Edward G.D. Tuddenham, M.D., Ph.D., a pioneer in the field of blood coagulation and a study co-author.

"This is a potentially life-changing treatment for patients with this disease and an important milestone for the field of gene therapy. It could have ramifications for the treatment of hemophilia A, other protein and liver disorders and chronic diseases such as cystic fibrosis," said first author Amit Nathwani, M.D., Ph.D., a faculty member at the UCL Cancer Institute, Royal Free Hospital and NHS Blood and Transplant (NHSBT).

Hemophilia B is caused by an inherited mistake in the gene for making a protein called Factor IX, which is essential for normal blood clotting. The gene is carried on the X chromosome. As a result, hemophilia B is almost exclusively a disease of men. About 1 in 30,000 individua
'/>"/>

Contact: Carrie Strehlau
media@stjude.org
901-595-2295
St. Jude Children's Research Hospital
Source:Eurekalert

Page: 1 2 3

Related medicine news :

1. Nicotine replacement therapy is over-promoted since most ex-smokers quit unassisted
2. Social Anxiety and Panic - Alternative Treatment to Drugs and Therapy
3. Longview Therapy Center, PLLC, Unveils Its Definition Of Hope
4. Short-term radiation therapy successful on breast cancer
5. ASCROs Response to NY Times Articles on Radiation Therapy Incidents
6. Children With Cerebral Palsy Benefitting From New Physical Therapy Regimen
7. DavisPTnetwork Partners with the New York Physical Therapy Association to Provide Online Continuing Education
8. Behavioral therapy improves sleep and lives of patients with pain
9. WHI data confirm short-term heart disease risks of combination menopausal hormone therapy
10. Promising therapy for relapsing multibple sclerosis
11. Split-course palliative radiotherapy confirmed as effective treatment for advanced NSCLC
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/24/2014)... 2014 Epigenetics finds applications in a ... oncology, drug discovery, developmental biology, and research for metabolic ... of the most widely used techniques for the discovery ... prevalence of cancer is rapidly growing worldwide. In 2012, ... cancer deaths, and 32.6 million people living with cancer ...
(Date:12/24/2014)... New studies reveal that women under the ... depression (March 6, 2014, JAMA Otolaryngology*). And although many people ... 50 percent according to recent polls) facing hearing loss head ... person feels and relates to the world. People who have ... made by way of:, , self-confidence , ...
(Date:12/24/2014)... 25, 2014 Today, Yunx.co.uk, a hot ... dresses and other special occasion outfits, unveils 65 ... are offered at discounted prices, up to 80% off. ... ladies with their fashionable designs, gorgeous looks and delicate ... The business is devoted to designing and offering high-quality ...
(Date:12/24/2014)... HealthDay Reporter TUESDAY, Dec. 23, 2014 (HealthDay ... for one year will be allowed to donate blood in the ... 31-year ban on donations from men who have sex with men. ... to release a new draft guidance in early 2015 that would ... The FDA is changing its policy based on data from other ...
(Date:12/24/2014)... 24, 2014 (HealthDay News) -- Adults who experienced ... of migraine headaches, suggests a study published online ... "Childhood maltreatment can have long-lasting effects, like ... adulthood," study author Dawn Buse, director of behavioral ... City, said in a journal news release. ...
Breaking Medicine News(10 mins):Health News:Global Epigenetics Market Was Valued at $413.24 Million in 2014 - New Research Report by MarketsandMarkets 2Health News:Global Epigenetics Market Was Valued at $413.24 Million in 2014 - New Research Report by MarketsandMarkets 3Health News:Recent Studys Underscore the Fact That One of the Best Gifts to Give This Holiday Season is a Hearing Test 2Health News:Recent Studys Underscore the Fact That One of the Best Gifts to Give This Holiday Season is a Hearing Test 3Health News:Recent Studys Underscore the Fact That One of the Best Gifts to Give This Holiday Season is a Hearing Test 4Health News:Recent Studys Underscore the Fact That One of the Best Gifts to Give This Holiday Season is a Hearing Test 5Health News:Recent Studys Underscore the Fact That One of the Best Gifts to Give This Holiday Season is a Hearing Test 6Health News:Yunx.co.uk Unveils 65 New Bridesmaid Dresses for the UK market 2Health News:FDA to Lift Ban on Blood Donations by Gay Men 2Health News:FDA to Lift Ban on Blood Donations by Gay Men 3Health News:Abuse in Childhood Tied to Migraines in Adulthood 2
... News) -- The incidence of head and neck cancers related ... States, with the greatest increase among middle-aged white men, a ... risk factors for head and neck cancers, but the sexually ... well, according to researchers from the Louisiana State University Health ...
... Long-term follow-up of a phase II clinical trial ... non-small cell lung cancer treated with belagenpumatucel-L, a therapeutic ... Annual Meeting 2012, held March 31 - April 4. ... to show unusually long survival in some patients," said ...
... Awakening from anesthesia is often associated with an ... restoration of awareness and orientation to one,s surroundings. ... consciousness emerges first. Using brain imaging techniques in ... Adjunct Professor Harry Scheinin, M.D. from the University ...
... (Boston) - Anurag Singh, PhD, assistant professor in ... Boston University School of Medicine has been invited ... for colon cancer at the American Association of ... Singh,s seminar, scheduled for Tuesday April 3rd, will ...
... , TUESDAY, April 3 (HealthDay News) -- Weight ... girls, self-esteem, according to a new study. "We found ... of obesity continued to see themselves as fat, despite changes ... associate professor of sociology at Purdue University in West Lafayette, ...
... Removing a lipoprotein receptor known as SR-BI may help ... presented at the American Association for Cancer Research Annual ... In vitro and mouse studies revealed that depletion of ... cell growth. SR-BI is a receptor for high-density ...
Cached Medicine News:Health News:HPV-Related Head, Neck Cancers on the Rise 2Health News:Scientists solving the mystery of human consciousness 2Health News:Scientists solving the mystery of human consciousness 3Health News:For Many Girls, Slimming Down Doesn't Help Self-Esteem 2Health News:Eliminating the 'good cholesterol' receptor may fight breast cancer 2
(Date:12/24/2014)...  Commonwealth Cornerstone Group (CCG) today announced the completion of ... to help fund the expansion and consolidation of healthcare services ... . PHN is the largest federally ... It is the largest employer in Sharon,s ... staff. Currently, PHN Sharon offers scattered services out ...
(Date:12/24/2014)... 2014 Soligenix, Inc. (OTCQB: SNGX) (Soligenix ... products that address unmet medical needs in the ... that on December 24, 2014 it closed its ... Company raised approximately $2.3 million in gross proceeds ... net proceeds from the offering to fund its ...
(Date:12/24/2014)... FLINT, Mich. , Dec. 24, 2014  Diplomat ... largest independent specialty pharmacy, is proud to announce ... and quality at Diplomat, will take office as the ... 2015. The formal induction ceremony will take place Feb. ... which will be held at the Renaissance Center in ...
Breaking Medicine Technology:Commonwealth Cornerstone Group Announces $8 Million in Tax Credits to Expand and Consolidate Healthcare Services in Sharon 2Commonwealth Cornerstone Group Announces $8 Million in Tax Credits to Expand and Consolidate Healthcare Services in Sharon 3Soligenix Announces Closing of Public Offering of Units 2Soligenix Announces Closing of Public Offering of Units 3Diplomat's senior director of education and quality to be inducted as 2015 president of the Michigan Pharmacists Association 2Diplomat's senior director of education and quality to be inducted as 2015 president of the Michigan Pharmacists Association 3
... Sponsor of ZENPEP(R) CF Cycle for Life , ... , May 3 Thousands of cyclists will take to the streets this summer ... the country to support research to find new therapies and a cure for cystic fibrosis. The ... June 26 . , , , ...
... May 2 Althea Technologies, Inc., ... and manufacturing announced today that it has acquired ... The IP portfolio includes, among other things, Altus, ... injectable technologies.  Applications of this protein crystal technology ...
Cached Medicine Technology:Cystic Fibrosis Foundation Launches National Cycling Series to Benefit Critical Research and Care 2Cystic Fibrosis Foundation Launches National Cycling Series to Benefit Critical Research and Care 3Althea Technologies Acquires Altus Pharmaceuticals'(R) Assets and IP Portfolio, Establishing Technology Platform for Protein Formulations. 2
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
... reference laboratory and a worldwide leader in ... an extensive test menu of highly complex ... anatomic pathology. Owned by the University of ... half of the nation's university teaching hospitals ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... reference laboratory and a worldwide leader in ... an extensive test menu of highly complex ... anatomic pathology. Owned by the University of ... half of the nation's university teaching hospitals ...
Medicine Products: