Navigation Links
UD researchers discover promising technique for repairing gene that causes spinal muscular atrophy
Date:2/12/2008

Researchers at the University of Delaware have discovered a novel technique--that acts like a spell-checker for correcting a misspelling in the DNA code--to repair the defective gene that causes spinal muscular atrophy (SMA). This hereditary neuromuscular disease is the number-one genetic killer of children under two years old.

Babies born with Type 1 SMA, the most severe form of the disease, can't walk, crawl, sit unsupported, lift their heads, or breathe normally. Fifty percent die before their second birthday.

The research is published in the Jan. 14 online edition of Experimental Cell Research. The study was supported by $477,500 in National Tobacco Settlement funds to the state of Delaware. The research grant was awarded through the Delaware Health Fund.

Think of it like a spell-check program--we're erasing the wrong letter in the DNA code and putting the right one in, said Eric Kmiec, professor of biological sciences at UD.

Kmiec, who holds 14 patents for gene-editing technologies at the University, collaborated with research scientist Darlise DiMatteo and undergraduate Stephanie Callahan on the discovery in his laboratory at the Delaware Biotechnology Institute.

The technique has shown promising results in tests in mice and is now poised for development by OrphageniX Inc., based in Wilmington, Del. The start-up company was incorporated in 2005 to commercialize UD-patented technologies for repairing genes that cause rare, hereditary, orphan diseases, so named because they have not been adopted by the pharmaceutical industry for the development of treatments.

According to the Families of Spinal Muscular Atrophy, an international, nonprofit organization, the disease affects one in 6,000 babies born, and one in 40 people is a genetic carrier.

A genetic 'bandage'

Spinal muscular atrophy is caused by a mutation in the SMN1 gene, which affects the motor neurons, the nerve cells
'/>"/>

Contact: Tracey Bryant
tbryant@udel.edu
302-831-8185
University of Delaware
Source:Eurekalert  

Page: 1 2 3 4

Related medicine news :

1. Researchers at BMC find disability does not necessarily follow disease in living to old age
2. Oregon researchers discover a mechanism leading to cleft palate
3. Penn childhood obesity researchers panelists at AAAS Town Hall Event
4. Researchers at Pulmonary Associates to study airway bypass procedure for severe emphysema
5. European blood researchers receive award to pursue research opportunities in US
6. SfN releases Best Practices for Protecting Researchers and Research
7. Researchers discover new battleground for viruses and immune cells
8. Researchers find less invasive, highly accurate methods
9. MSU researchers make new discoveries on what does and doesnt affect immune system
10. Researchers query FDA about updating requirements for drug approval
11. MITs McGovern Institute researchers awarded $8.5M to study brain basis of autism and dyslexia
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
UD researchers discover promising technique for repairing gene that causes spinal muscular atrophy
(Date:9/17/2014)... September 17, 2014 Dr. Shervin Naderi, ... surgeons, has been recognized as a recipient of the 2014 ... only the best of the best as it honors physicians ... Dr. Naderi, who is affectionately known by his plastic surgeon ... handful of true rhinoplasty specialist surgeons in the ...
(Date:9/17/2014)... Steven Reinberg HealthDay Reporter ... The number of Americans dying from accidental overdoses of ... health officials reported Tuesday. Deaths from overdoses of ... climbed from 1.4 per 100,000 people to 5.4 ... Control and Prevention. That means about 3,000 people ...
(Date:9/17/2014)... one can say whether yoga provides clinical benefits to ... in the Journal of Psychiatric Practice , researchers ... with the condition who practice yoga. What the collective ... help, but it sometimes carries risks, too. , "There ... disorder," said lead author Lisa Uebelacker, associate professor (research) ...
(Date:9/17/2014)... (PRWEB) September 17, 2014 A  recent ... of a 24-year-old man named Advait Thakur who had ... caused by degenerative disc disease. This disease led to ... his nerve roots and causing severe pain. Leading spine ... and how it can lead to such severe pain, ...
(Date:9/17/2014)... 2014 The disability rights group Not Dead ... vigil against the World Federation of Right to Die Societies ... American group Final Exit Network is hosting the meeting, which ... group has scheduled a rally to open the protest vigil ... PM. , ”We are here to contradict the message of ...
Breaking Medicine News(10 mins):Health News:Vitals Honors Dr. Shervin Naderi With Top 10 Doctor Award 2Health News:Vitals Honors Dr. Shervin Naderi With Top 10 Doctor Award 3Health News:Deaths From Narcotic Painkillers Quadrupled in Past Decade: CDC 2Health News:Deaths From Narcotic Painkillers Quadrupled in Past Decade: CDC 3Health News:Survey finds benefits, risks of yoga for bipolar disorder 2Health News:Survey finds benefits, risks of yoga for bipolar disorder 3Health News:24-Year-Old Back in College After Degenerative Disc Disease Kept Him Out 2Health News:24-Year-Old Back in College After Degenerative Disc Disease Kept Him Out 3Health News:Disability Rights Activists to Protest International Euthanasia Group Meeting in Chicago 2
... VILLAGE, Calif., Feb. 27 Riverview Medical,Center, ... recognized for service,excellence under the J.D. Power ... a strong commitment by Riverview,Medical Center to ... (Logo: http://www.newscom.com/cgi-bin/prnh/20050527/LAF028LOGO-a ), The service ...
... BOSTON, Feb. 27 Although,physician-scientists and supplement manufacturers ... sparring over multivitamins. In fact, half the,physicians on ... taking a,multivitamin themselves. In recent years, Harvard Men,s ... that even if they don,t,help, they won,t hurt. ...
... The Association for Professionals in Infection Control and ... selected by Blue Shield of California Foundation (BSCF) ... healthcare-associated infections (HAIs). , APIC will join BSCF ... Foundations innovative California Healthcare-Associated Infection Prevention Initiative to ...
... Arthritis Foundation to Launch the ,I ... Can with RA in the Kitchen(TM), Campaign, NEW ... approach to cooking, which involves using 70 percent,store-bought items and ... millions of busy Americans. Unknown to most, Lee,s signature,style was ...
... Feb. 27 /PRNewswire-FirstCall/ - (Imaging Dynamics Company,Ltd.) (TSX: ... image,quality and value, today announced that it was ... digital radiography system. This is the 7th,U.S. patent ... awarded to IDC in,countries around the world for ...
... that is an early indicator of kidney dysfunction in ... of Georgia researchers have found. , Microalbuminuria, excessive amounts ... among diabetics, says Dr. Gregory Harshfield, director of the ... reason, the few studies that have looked at adolescent ...
Cached Medicine News:Health News:J.D. Power and Associates Reports: Riverview Medical Center Recognized for Providing an Emergency Department Experience 2Health News:J.D. Power and Associates Reports: Riverview Medical Center Recognized for Providing an Emergency Department Experience 3Health News:It's Time to Reassess the Value, Safety of Multivitamin Use, Says Harvard Men's Health Watch 2Health News:Celebrity Chef Sandra Lee Offers Recipes and Shortcuts in the Kitchen to People with Rheumatoid Arthritis 2Health News:Celebrity Chef Sandra Lee Offers Recipes and Shortcuts in the Kitchen to People with Rheumatoid Arthritis 3Health News:Celebrity Chef Sandra Lee Offers Recipes and Shortcuts in the Kitchen to People with Rheumatoid Arthritis 4Health News:Celebrity Chef Sandra Lee Offers Recipes and Shortcuts in the Kitchen to People with Rheumatoid Arthritis 5Health News:Celebrity Chef Sandra Lee Offers Recipes and Shortcuts in the Kitchen to People with Rheumatoid Arthritis 6Health News:IDC receives patent on dual energy imaging technology 2Health News:IDC receives patent on dual energy imaging technology 3Health News:Urine protein may be present before hypertension diagnosis in at-risk adolescents 2
(Date:9/17/2014)... , Sept. 17, 2014 Isis ... that it has earned a $4 million milestone ... ) associated with the initiation of a Phase ... multi-drug resistant (MDR), gram-negative bacterial infections.  The Phase ... to evaluate the efficacy and safety of plazomicin ...
(Date:9/17/2014)... Sept. 17, 2014 Sutro Biopharma, a biopharmaceutical ... the biopharmaceutical division of Merck KGaA, Darmstadt, ... the United States and ... license agreement to develop antibody drug conjugates (ADCs). ADCs ... drug. The antibody is thought to specifically target and ...
(Date:9/17/2014)... Pa. , Sept. 17, 2014  Fortune Oil ... officially changed its name to Manzo Pharmaceuticals, Inc. with ... to take effect today, September 17, 2014. Also taking ... The approval is a pivotal event in ... . Mr. Manzo explained, "We could not proceed with ...
Breaking Medicine Technology:Isis Pharmaceuticals Earns $4 Million from Achaogen for the Initiation of a Phase 3 Study of Plazomicin 2Isis Pharmaceuticals Earns $4 Million from Achaogen for the Initiation of a Phase 3 Study of Plazomicin 3Sutro Biopharma and Merck KGaA of Darmstadt, Germany, to Partner on Development of Antibody Drug Conjugates 2Sutro Biopharma and Merck KGaA of Darmstadt, Germany, to Partner on Development of Antibody Drug Conjugates 3Sutro Biopharma and Merck KGaA of Darmstadt, Germany, to Partner on Development of Antibody Drug Conjugates 4Fortune Oil and Gas, Inc. announces 1 for 10 reverse split along with a name change to Manzo Pharmaceuticals is now effective 2Fortune Oil and Gas, Inc. announces 1 for 10 reverse split along with a name change to Manzo Pharmaceuticals is now effective 3
... 3, 2011 Coast Dental Services, Inc., announced today ... the assets of Dental Technology, Inc. (DTI), doing business ... licensed  health care service plan. DTI and its subsidiaries ... Texas, employing 1,200 staff members, including 130 dentists.  The ...
... 3, 2011 The Food and Drug Administration says ... retail outlets that falsely claim to treat, prevent, or ... you’re taking a big risk unless you see a ... (Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO ) View a ...
Cached Medicine Technology:Coast Dental to Acquire the Assets of SmileCare 2
... Accu-Temp Cauteries provide a ... for cauterization in a wide ... single use cautery is packaged ... cap to lock switch in ...
... provide a broad selection of tip ... variety of ophthalmic procedures. Each single ... with a safety cap to lock ... when not in use, and carries ...
... Cauteries provide a broad selection of ... wide variety of ophthalmic procedures. Each ... complete with a safety cap to ... position when not in use, and ...
... Luminary LASIK Light Probes provide the surgeon ... the corneal interface to be easily visualized ... the Tindal effect and light scatter technique, ... debris, epithelial deposits, flap edge folds, corneal ...
Medicine Products: