All posts by: Michael Zand

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Does CRISPR cause unintended mutations? Authors of controversial study reverse their findings – Genetic Literacy Project

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2018-03-27 NEWS

Genetic Literacy Project

Does CRISPR cause unintended mutations? Authors of controversial study reverse their findings
Genetic Literacy Project
[Editor’s note: The authors of a controversial report in Nature in May 2017 suggested that the popular CRISPR gene-editing technique could result in hundreds of unintended mutations. It was received with skepticism by scientists but anti-biotechnology

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2018-03-27 NEWS

GlobeNewswire (press release)

AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1
GlobeNewswire (press release)
CHICAGO, March 27, 2018 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that Japan’s

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2018-03-27 NEWS

Times of Malta

Who should pay for million-dollar gene therapies?
Times of Malta
If you were born with a rare form of blindness, there is now a treatment for you that may restore your eyesight. That’s because gene therapies became a clinical reality in 2017. Yet many people with rare diseases that could be treated in this way may

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2018-03-27 NEWS

PMLiVE

Takeda’s Alofisel becomes Europe’s first allogenic stem cell therapy
PMLiVE
Takeda has chalked up a big win this week after receiving a European licence for Alofisel (darvadstrocel), marking the first approved allogenic stem cell therapy set to hit the EU market. Alofisel – previously Cx601 – is approved to treat complex