Krystof bankiewicz - Latest News Update, Information, Photos & Videos on Krystof bankiewicz at Lokmat Times

Researchers recently developed a novel method of gene therapy that can help children born with a rare genetic disorder called AADC deficiency. This deficiency causes severe physical and developmental disabilities.Research findings were published online in the journal Nature Communications. The study, led by researchers at The Ohio State University Wexner Medical Center and The Ohio State University College of Medicine, offers new hope to those living with incurable genetic and neurodegenerative diseases.This study describes the findings from the targeted delivery of gene therapy to the midbrain to treat a rare deadly neurodevelopmental disorder in children with a neurogenetic disease, aromatic L-amino acid decarboxylase (AADC) deficiency characterized by the deficient synthesis of dopamine and serotonin.Only about 135 children worldwide are known to be missing the enzyme that produces dopamine in the central nervous system, which fuels pathways in the brain responsible for motor function and emotions. Without this enzyme, children lack muscle control and are usually unable to speak, feed themselves, or even hold up their heads. They also suffer from seizure-like episodes called oculogyric crises that can last for hours."Remarkably, these episodes are the first symptom to disappear after gene therapy surgery, and they never return," said study co-author Dr. Krystof Bankiewicz, professor of neurological surgery at Ohio State College of Medicine who leads the Bankiewicz Lab. "In the months that follow, many patients experience life-changing improvements. Not only do they begin laughing and have improved mood, but many are able to begin speaking and even walking. They are making up for the time they lost during their abnormal development."The directed gene therapy in seven children ages 4 to 9 who were infused with the viral vector resulted in dramatic improvement of symptoms, motor function and quality of life. Six children were treated at UCSF Benioff Children's Hospital in San Francisco and one at Ohio State Wexner Medical Center. This therapeutic modality promises to transform the treatment of AADC deficiency and other similar disorders of the brain in the future, Bankiewicz said.During gene therapy surgery, physicians infuse a benign virus programmed with specific DNA into precisely targeted areas of the brain. The infusion is delivered extremely slowly as surgeons monitor exactly how it spreads within the brain using real-time MRI imaging."Really, what we're doing is introducing a different code to the cell," said Dr. James "Brad" Elder, director of neurosurgical oncology at Ohio State Wexner Medical Center's Neurological Institute. "And we're watching the whole thing happen live. So, we continuously repeat the MRI and we can see the infusion blossom within the desired nucleus."Researchers believe this same method of gene therapy can be used to treat other genetic disorders as well as common neurodegenerative diseases, such as Parkinson's and Alzheimer's disease. Clinical trials are underway to test this procedure in others living with debilitating and incurable neurological conditions.The directed gene therapy, in these patients, resulted in dramatic improvement of symptoms, motor function and quality of life. This therapeutic modality promises to transform the treatment of AADC deficiency and other similar disorders of the brain in the future.( With inputs from ANI )Disclaimer: This post has been auto-published from an agency feed without any modifications to the text and has not been reviewed by an editor

Home

City

Latest News

Cricket

LT Premium

National

International

Maharashtra

Opinions

Chhatrapati Sambhajinagar

Nagpur

Entertainment

Photos

Business

Technology

Sports

Lifestyle

Social Viral

Lokmat Games

Health

Pahalgam Terror Attack

Tahawwur Rana

IPL 2025

CM Devendra Fadnavis

Mumbai Local

Weather Update

Krystof BankiewiczFOLLOW

Krystof bankiewicz, Latest News

Technology :Study develops novel gene therapy method to help children with neurological deficiencies

Researchers recently developed a novel method of gene therapy that can help children born with a rare genetic disorder called AADC deficiency. This deficiency causes severe physical and developmental disabilities. ...