September 27, 2020         
Celebrity Nitzia Chama Will Host Launch Event For KUL CBD Luxury Skincare At Curacao Department Stores   •   Censorship and the Dangers of Being Silenced   •   Curative Researchers Initiate Research Study to Test Efficacy of Self-Collected COVID-19 Tests   •   The Return of the Pope of Buddhism Scepter by His Holiness Dorje Chang Buddha III was Rejected   •   U.S. Women's Chamber of Commerce Endorses Joe Biden   •   Frog Street Offers Extensive "At-Home Learning" Resources to Help Children Stay Engaged in New Hybrid Learning Environments   •   Pres. Donald Trump to speak on Friday Night at Family Research Council Action's Values Voter Summit 2020   •   Association of Independent Mortgage Experts Partners with United Wholesale Mortgage and Home Point Financial to Introduce Small   •   LegalShield Leadership Convention, Lead the Change, to Bring Record Number of Associates Together Virtually   •   TherapeuticsMD Provides Update on Third Quarter Progress   •   ERLC President Russell Moore Affirms Amy Coney Barrett as SCOTUS Justice Nominee   •   Everything You Need To Know About PEOPLE EN ESPAÑOL's Festival 2020 Returning Virtually!   •   In response to Governor Newsom signing Assembly Bill 2149 into law Postmates along with other concerned parties, Dine Black LA a   •   Laird Superfood Announces Closing of Initial Public Offering and Exercise in Full of the Underwriters’ Option to Purchase   •   Dow leaders recognized on 2020 HERoes Women Role Model lists   •   C-Sweet Webinar: “How We Can Make Difference” Part Three in a Series on Why Diversity Matters   •   AHF Rings Alarm Over Nationwide Shortage of STD Test Kits   •   ADEA Statement in Support of Diversity, Equity and Inclusion Workplace Training   •   Books-A-Million Honors U.S. Military with Coffee for the Troops Program Through October 24   •   Cubic Introduces New Ventra Mobile App for Chicagoland Travelers
Bookmark and Share

Rosalind Franklin University Researcher Awarded NIH Grant For Promising Pediatric Genetic Disease Therapeutic Approach

Rosalind Franklin University Researcher Awarded NIH Grant For Promising Pediatric Genetic Disease Therapeutic Approach

PR Newswire

NORTH CHICAGO, Ill., July 31, 2020 /PRNewswire/ -- Researchers led by a team at Rosalind Franklin University of Medicine and Science have devised a new therapeutic approach to treating a rare but deadly neurodegenerative genetic disease in children. The study, published this week in Nature Medicine, addresses the urgent need to discover treatments for Batten disease, a fatal neurodegenerative disorder affecting up to four of every 100,000 children in the United States. The disease has no cure, and treatment options are few.

Professor Michelle Hastings, PhD, director for RFU's Center for Genetic Diseases, led the study in collaboration with researchers at Ionis Pharmaceuticals and Sanford Research. Published July 27, "Therapeutic Efficacy of Antisense Oligonucleotides in Mouse Models of CLN3 Batten Disease" demonstrates that symptoms in rodent models with the disease can be treated through the application of antisense oligonucleotides (ASOs) designed to attach specifically to the CLN3 gene product disrupted in CLN3 Batten disease. The researchers also demonstrated that human cells cultured from patients with the disease respond to treatment in a manner that indicates a reversal of cellular defects.

"When rodents were treated with the therapeutic CLN3 corrector, their lifespan was extended, motor skills improved and waste buildup in the brain was lessened," said Dr. Hastings. "It was a surprising demonstration that partial correction of the defective CLN3 gene can lead to meaningful improvements in an animal model with this disease."

"Our work tested a novel approach to therapeutically target the expression of the most common cause of the disease using ASOs — directed to the mutated form of the gene," said graduate student Jessica Centa, leading author on the paper. "These results are a critical step toward our long-term goal of developing a treatment for CLN3 Batten disease."

The FDA has approved a number of antisense-based therapeutics for genetic diseases in the last few years, including spinal muscular atrophy, another pediatric neurodegenerative disorder. The number of genetic diseases is estimated at more than 7,000, and most have few if any effective treatments. ASO technology offers a potential therapeutic answer to this problem.

"We have work to do before this therapeutic will be ready to test in humans, but the animal studies suggest that our approach may provide an effective treatment in patients," Dr. Hastings said.

Dr. Hastings was recently awarded a $2.3 million grant from the National Institute of Neurological Disorders and Stroke to advance the team's investigation into correcting faulty gene expression associated with CLN3 Batten disease using antisense oligonucleotide technology. ASO is a therapeutic platform with potential applications for a large number of inherited genetic diseases. The grant was awarded in collaboration with colleagues in the Pediatrics and Rare Diseases group at Sanford Research, Sioux Falls, SD, led by neuroscientist Jill Weimer, PhD. 

The Center for Genetic Diseases is one of six disease-focused centers within RFU's new Innovation and Research Park that are driving the translation of novel medical discoveries through expanded collaborations with industry and biotech entrepreneurs.

"Dr. Hastings' latest NIH grant is an acknowledgement of her team's seminal work to find therapeutic options for a devastating pediatric neurodegenerative disease and also RFU's strong culture of collaboration, innovation and mentorship," said Executive Vice-President for Research Ronald Kaplan, PhD. "We look forward to the advancement of this study, which offers hope for a healthier future to so many children and their families."

About Rosalind Franklin University

Rosalind Franklin University of Medicine and Science is committed to serving the population through the interprofessional education of health and biomedical professionals and the discovery of knowledge dedicated to improving wellness. The university embodies the spirit of inquiry and excellence modeled by its namesake Dr. Rosalind Franklin, whose Photo 51 was crucial to solving the structure of DNA. In 2020, the university commemorates the 100th anniversary of the scientist's birth. Recognized for its research in areas including neuroscience, brain-related diseases, inherited disorders, diabetes, obesity, and gait and balance, RFU encompasses the Chicago Medical School, College of Health Professions, College of Pharmacy, School of Graduate and Postdoctoral Studies and the Dr. William M. Scholl College of Podiatric Medicine. Learn more at rosalindfranklin.edu

Office of Marketing and Communications

dan.moran@rosalindfranklin.edu  

Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/rosalind-franklin-university-researcher-awarded-nih-grant-for-promising-pediatric-genetic-disease-therapeutic-approach-301104040.html

SOURCE Rosalind Franklin University of Medicine and Science



Back to top
| Back to home page
Video

White House Live Stream
LIVE VIDEO EVERY SATURDAY
alsharpton Rev. Al Sharpton
9 to 11 am EST
jjackson Rev. Jesse Jackson
10 to noon CST


Video

LIVE BROADCASTS
Sounds Make the News ®
WAOK-Urban
Atlanta - WAOK-Urban
KPFA-Progressive
Berkley / San Francisco - KPFA-Progressive
WVON-Urban
Chicago - WVON-Urban
KJLH - Urban
Los Angeles - KJLH - Urban
WKDM-Mandarin Chinese
New York - WKDM-Mandarin Chinese
WADO-Spanish
New York - WADO-Spanish
WBAI - Progressive
New York - WBAI - Progressive
WOL-Urban
Washington - WOL-Urban

Listen to United Natiosns News