HOME >> BIOLOGY >> NEWS
Novel gene therapy on the horizon, says Brenner Children's Hospital pediatrician

WINSTON-SALEM, N.C. Researchers at Brenner Children's Hospital are working on a novel gene therapy to help new moms and their babies who suffer from genetic defects, according to findings reported at the Society of Pediatric Research in Seattle today.

R. Mark Payne, M.D., a pediatric cardiologist and researcher at Brenner Children's Hospital, has discovered a new way to transmit proteins across cell membranes a finding that in the future might prevent or treat diseases like Sudden Infant Death Syndrome (SIDS) and Trifunctional Protein (TFP) deficiency in infants and their mothers.

"The treatment involves getting the right protein into the cells or mitochondria they need to function properly," Payne said. "Mitochondria produce most of the energy within cells and defects in their function are among the most common genetic defects in the United States and currently can not be prevented."

Some genetic defects occur when mitochondria lack a certain protein, which cause babies, and in some cases their mothers, to become sick and die, Payne said. Current gene therapy attempts have tried injecting the patient with a virus that corrects the defect. However, the effectiveness of this approach is very poor and the side effects aren't good patients may die from the treatments, according to Payne.

"Instead of injecting a virus into the cells, we want to take the needed protein and inject only that protein into the cells," Payne said. "This is highly efficient technology the needed proteins get absorbed and are positioned correctly within the mitochondria located in the cell, which is necessary to correct the defect."

Payne and his colleagues say this new technology could mean that mothers suffering from genetic abnormalities such as TFP may be able to receive injections until their due date preventing premature delivery and sometimes death for both the mother and infant.

TFP is one of many genetic defects that involve mitochondria
'"/>

Contact: Rae Beasley
rabeasle@wfubmc.edu
336-716-6878
Wake Forest University Baptist Medical Center
4-May-2003


Page: 1 2

Related biology news :

1. Novel IBD therapeutic approaches reported from Washington Univ., Barcelona, LSU at APS meeting
2. Novel therapeutic bortezomib moves to phase II trial in lung cancer patients
3. Novel approaches to current cellular therapies continue progress toward disease prevention
4. Novel therapies show promise against myeloid leukemia
5. Novel sensors help clear the air
6. Fox Chase Cancer Center study: Novel DNA-repair gene mutation can cause resistance to cancer drugs
7. Novel proteins designed that block inflammation regulator associated with rheumatoid arthritis
8. Novel therapeutic target identified in fight against Rheumatoid Arthritis
9. Novel bacterium detoxifies chlorinated pollutants
10. Novel flu vaccine shows promise in mice
11. Novel method identifies hidden genes

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:8/14/2019)... TORONTO (PRWEB) , ... August 14, 2019 , ... Join ... live session on Tuesday, September 10, 2019 at 1pm EDT to learn ... stratification and effective management. , NAFLD is the most common diffuse liver disease, ...
(Date:8/14/2019)... Calif. (PRWEB) , ... August 13, 2019 , ... Dr. Julie Reck of Veterinary ... to her patients. Her first stem cell patient, her own geriatric Australian Shepherd, ... According to Dr. Reck, Simon wanted to play but his body was weak, and he ...
(Date:8/6/2019)... (PRWEB) , ... August 05, 2019 , ... Keck Graduate ... KGI School of Pharmacy and Health Sciences (SPHS). , “Dean Zdanowicz is highly regarded ... of Trustees Chairman Jim Widergren. “He is a great choice to build on the ...
Breaking Biology News(10 mins):
(Date:7/9/2019)... ... July 08, 2019 , ... Today, at the BIO World ... Corn Growers Association (NCGA) announced the winners of the Consider Corn Challenge II. ... process using field corn to produce biobased materials. , “Corn is a sustainable, ...
(Date:6/18/2019)... ... June 18, 2019 , ... Personalized Stem Cells, Inc ... application to the FDA for use of a person’s own adipose-derived stem cells to ... osteoarthritis in the knee. This IND is the first of several planned clinical ...
(Date:6/11/2019)... ... June 11, 2019 , ... Mytonomy, the ... national leader in opioid treatment, announced today they are partnering to combat the ... recently deployed its virtual care platform at the Mayo Clinic to drive shared ...
(Date:5/31/2019)... ... May 29, 2019 , ... ... applications from the USPTO providing proprietary interest to our methodology, processes, and ... DISORDER DIAGNOSIS AND TREATMENT… extends Somnology’s IP rights including our proprietary sleep ...
Breaking Biology Technology:
Cached News: