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CRISPR NIH Director’s Blog

Posted on September 11th, 2018 by Dr. Francis Collins Caption: A CRISPR/cas9 gene editing-based treatment restored production of dystrophin proteins (green) in the diaphragm muscles of dogs with Duchenne muscular dystrophy.Credit: UT Southwestern CRISPR and other gene editing tools hold great promise for curing a wide range of devastating conditions caused by misspellings in DNA

This New, Cutting-Edge Treatment Could Be the End of Baldness – Reader’s Digest

docent/ShutterstockWhether or not theres a scientific benefit to being baldwell let the follically challenged among us be the judge of thatscientists continue to search for a balding cure. According to UCLA researchers, that isnt completely out of the question.

UCLA scientists identify a new way to activate stem cells to make hair grow – UCLA Newsroom

UCLA researchers have discovered a new way to activate the stem cells in the hair follicle to make hair grow. The research, led by scientists Heather Christofk and William Lowry, may lead to new drugs that could promote hair growth for people with baldness or alopecia, which is hair loss associated with such factors as hormonal imbalance, stress, aging or chemotherapy treatment. The research was published in the journal Nature Cell Biology

CRISPR Helped ‘Edit’ A Human Embryo But That’s Not All It Can Do – HuffPost

The results of the first gene-editing experiment conducted on a human embryo in the United States were published on Wednesday, and their implications cant be overstated. A research team was able to erase the gene mutation responsible for a condition causing sudden heart failure from a one-cell human embryo

Donor appeal for poorly toddler ‘may have saved other lives’ – Bicester Advertiser

COUNTLESS lives across the world could be saved by an Oxfordshire familys appeal to find a bone marrow donor for their little boy.

Oxford University staff join bone marrow stem cell donor drive for Oxford toddler Ally Kim – Witney Gazette

COUNTLESS lives across the world could be saved by an Oxfordshire familys appeal to find a bone marrow donor for their little boy. Two-year-old Alastair Ally Kim has Chronic Granulomatous Disorder (CGD), a life-threatening condition.

Oxford University staff join bone marrow stem cell donor drive for … – Oxford Mail

COUNTLESS lives across the world could be saved by an Oxfordshire familys appeal to find a bone marrow donor for their little boy.

Donor appeal for poorly toddler ‘may have saved other lives’ – Witney Gazette

COUNTLESS lives across the world could be saved by an Oxfordshire familys appeal to find a bone marrow donor for their little boy. Two-year-old Alastair Ally Kim has Chronic Granulomatous Disorder (CGD), a life-threatening condition.

Search goes on for bone marrow match for little Longworth lad … – Oxford Mail

ANDREW and Judy Kim are still searching the globe for a donor for their two-year-old son after he was diagnosed with a rare genetic condition. The couple's son Alastair was diagnosed with chronic granulomatous disorder (CGD) in February last year. Mr and Mrs Kim launched an appeal for help in September but the search is still on for a matching donor and their son still needs hospital treatment.

Research and Markets: Acute Spinal Cord Injury – Pipeline Review Report, H1 2013 Edition

DUBLIN--(BUSINESS WIRE)-- Research and Markets (http://www.researchandmarkets.com/research/hls5jn/acute_spinal_cord) has announced the addition of the "Acute Spinal Cord Injury - Pipeline Review, H1 2013" report to their offering. 'Acute Spinal Cord Injury - Pipeline Review, H2 2013', provides an overview of the indication's therapeutic pipeline.

Research and Markets: Neuroprotection – Drugs, Markets and Companies – 2013-2022

DUBLIN--(BUSINESS WIRE)-- Research and Markets (http://www.researchandmarkets.com/research/d2p5hw/neuroprotection) has announced the addition of Jain PharmaBiotech's new report "Neuroprotection - Drugs, Markets and Companies" to their offering. This updated report now contains even more company profiles than the previous edition! This report describes the role of neuroprotection in acute disorders such as stroke and injuries of the nervous system as well as in chronic diseases such as neurodegenerative disorders because many of the underlying mechanisms of damage to neural tissues are similar in all these conditions and several products are used in more than one disorder.

SGEN/Astellas to Co-Develop Another ADC

Seattle Genetics (SGEN) and Agensys, a subsidiary of Astellas Pharma Inc. (ALPMY), recently announced that the former has exercised its option to co-develop one more antibody-drug conjugate (ADC), ASG-15ME, under an existing collaboration. ASG-15ME is an ADC which targets the tumor antigen SLITRK6.

SGEN Seeks Adcetris Label Expansion

Seattle Genetics, Inc.

Children improve in rare disorder with own stem cells

London, Feb 29 (IANS) Children shot with their own stem cells, for the very first time in a rare immune disorder, have shown improvement. The condition, known as X-CGD, is caused by faulty genes. Doctors were able to take a sample of the children's stem cells, manipulate them in the lab and reintroduce them.

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