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Families hope 'Frankenstein science' lobby will not stop gene cure for mitochondrial disease
Change to IVF rules could make Britain the first country to allow therapy to change DNA in embryosDeniz Safak was five years old when he first displayed symptoms of the disease that would later take his life. "He started being sick and had intense, stroke-like seizures," his mother, Ruth, recalled.Doctors were baffled by the boy's condition and it took months before a diagnosis was made. Ruth and her husband, Erdhal, were told that Deniz was suffering from mitochondrial disease, an incurable condition that is passed from mother to child and can often be fatal.Deniz's condition continued to worsen. By the time he died last ...
Source: Guardian Unlimited Science - February 15, 2014 Category: Science Authors: Robin McKie Tags: Embryos IVF Genetics Children Biology News Health Medical research Society UK news The Observer Science Source Type: news

Neuroscience is the Next Oncology
by Michael D. Ehlers, MD, PhD Dr. Ehlers is with Biogen in Cambridge, Massachusetts. Innov Clin Neurosci. 2018;15(3–4):15–16 Funding: No funding was received for the preparation of this article. Disclosures: Dr. Ehlers is an employee and shareholder at Biogen Inc. in Cambridge, Massachusetts. Prominent and expensive failures in Alzheimer’s disease therapies have led to a contagious belief system in some parts of the biopharma industry that neuroscience is just too hard, too risky, and too uncertain. But, might this belief system itself be a residual bias of the past? Close inspection reveals all the signs of a coming...
Source: Innovations in Clinical Neuroscience - April 1, 2018 Category: Neuroscience Authors: ICNS Online Editor Tags: Commentary Current Issue Source Type: research

Genetics of Dravet Syndrome and its Targeted Therapy by Nanomedicine: A Roadmap for Future Treatment of Drug Resistant Seizures
Curr Mol Pharmacol. 2022 Aug 19. doi: 10.2174/1874467215666220819143105. Online ahead of print.ABSTRACTAccording to the World Health Organization (WHO), epilepsy is the 4th most prevalent neurological disorder after migraine, stroke, and Alzheimer's disease. There are numerous types of epileptic syndrome that are reported in children; one of them is Dravet syndrome. It is a neurological disorder of infants' outset during the first year of life. Dravet syndrome is a genetically determined syndrome and the most studied form of genetic epilepsy. Nearly 70-80% of its cases are due to genetic alterations in the SCN1A gene, and ...
Source: Epilepsy Curr - August 20, 2022 Category: Neurology Authors: Muhammad Ikram Sufian Rasheed Source Type: research