Category: <span>Genetics</span>

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New treatment hope for those affected by muscle loss

by Wendy Davidson, University of Aberdeen New research from the University of Aberdeen has identified the genes responsible for muscle mass These findings may identify those most at risk from muscle loss as they get older and identifying these genes may unlock the potential for targeted drug therapies that could prevent further muscle loss. Published...

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Know thy mitochondria: Autoimmunity to organelles and their DNA

by John Hewitt , Medical Xpress The immune system uses its mitochondria to self-stimulate innate and adaptive responses to infection. Reactive oxygen species (ROS), immunogenic mitochondrial DNA (mtDNA), and even whole mitochondria are locally mobilized in a delicate balance to create hot spots of inflammatory action. When normal limiting feedback on these processes is compromised,...

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Using gene therapy to treat chronic traumatic encephalopathy

MARY ANN LIEBERT, INC./GENETIC ENGINEERING NEWS New Rochelle, NY, January 3, 2020–A new study shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE). The study, which demonstrated the effectiveness of direct delivery of gene therapy into the brain of a mouse model of CTE, is published in...

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What is Superman Syndrome?

By Hidaya Aliouche, B.Sc .Reviewed by Dr. Jennifer Logan, MD, MPH Superman syndrome, also known as 47, XYY, is a condition classified as a chromosomal aneuploidy (which is an abnormality in chromosome structure and/or number) in which males have an additional Y chromosome. The normal male sex chromosomes are XY; in the case of Superman syndrome,...

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New technology allows control of gene therapy doses

by  The Scripps Research Institute Scientists at Scripps Research in Jupiter have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing. The feat, reported in the scientific journal Nature Biotechnology, offers gene therapy designers what may be the first viable technique for adjusting the activity levels of...

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For CRISPR, tweaking DNA fragments before inserting yields highest efficiency rates yet

University of Illinois researchers achieved the highest reported rates of inserting genes into human cells with the CRISPR-Cas9 gene-editing system, a necessary step for harnessing CRISPR for clinical gene-therapy applications. By chemically tweaking the ends of the DNA to be inserted, the new technique is up to five times more efficient than current approaches. The...

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What is Superman Syndrome?

By Hidaya Aliouche, B.Sc.Reviewed by Dr. Jennifer Logan, MD, MPH Superman syndrome, also known as 47, XYY, is a condition classified as a chromosomal aneuploidy (which is an abnormality in chromosome structure and/or number) in which males have an additional Y chromosome. Image Credit: Rost9 / Shutterstock.com The normal male sex chromosomes are XY; in the case...

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Online tool helps patients demystify the ‘Pandora’s box’ of genomic sequencing

by  St. Michael’s Hospital A decision aid developed to support patients undergoing genomic sequencing can reduce the amount of time patients spend speaking with overburdened genetic counselors while helping them were more knowledgeable about the benefits of sequencing, suggests a study from St. Michael’s Hospital. Published in Genetics in Medicine, the official journal of the American...

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Gene therapy is facing its biggest challenge yet

After finally gaining traction as a potential treatment for certain genetic disorders, gene therapy tackles the challenge of sickle-cell disease. Grajevis Bakatunkanda’s mother knew the signs: when her son lost interest in dinner, that meant the pain was on its way. It would strike, like clockwork, nearly every week. Soon the shy, skinny boy would...

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EXPERTS HORRIFIED BY LEAKED CRISPR BABY STUDY

THE PAPER ACCOMPANYING THE CONTROVERSIAL EXPERIMENT WENT UNPUBLISHED — UNTIL NOW. BY KRISTIN HOUSER / DECEMBER 04 2019 Chinese scientist He Jiankui’s creation of the world’s first gene-edited human babies was undoubtedly one of the most impactful science stories of 2018. But for as much attention as the experiment received, the paper detailing it was never actually published — until now....