Category REGENERATIVE MEDICINE

Geneticists retract study suggesting first CRISPR babies might die early

Researchers rapidly corrected finding through discussions on social media and preprints. Source Nature A study that raised questions over the future health of the world’s first gene-edited babies has been retracted because of key errors that undermined its conclusion. The research, published in June 2019 in Nature Medicine1, had suggested that people with two copies of a natural genetic mutation

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Mila’s N-of-1 Trial Detailed in NEJM

Neurologic scores stabilized, seizures diminished with tailored antisense oligonucleotide therapy The researchers who developed a personalized antisense oligonucleotide (ASO) for a little girl with a form of Batten disease — all in record time — have detailed their case in the New England Journal of Medicine. Timothy Yu, MD, PhD, of Boston Children’s Hospital, and colleagues explained how

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RPE65 Gene Therapy Effective for Retinal Dystrophy

Subretinal administration of the gene therapy AAV2/5-OPTIRPE65 showed improvements in vision-guided mobility, retinal sensitivity, and foveal-driven visual function for patients with RPE65-associated retinal dystrophy, according to findings from a phase 1/2 study presented at the 2019 American Academy of Ophthalmology Annual Meeting. In the early-stage study, there was a significant change in the ability to complete a

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New articles on the ethics of genome editing published in The CRISPR Journal

Source http://www.liebertpub.com/ The CRISPR Journal announces the publication of its October 2019 Special Issue on The Ethics of Human Genome Editing. The Journal is led by Editor-in-Chief Rodolphe Barrangou, PhD (North Carolina State University) and Executive Editor Dr. Kevin Davies. For full-text copies of articles or to arrange interviews with Dr. Barrangou, Dr. Davies, authors, or

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Overcoming the barriers to cell and gene therapies in Europe. BIA and ARM position

By Annie Hubert, Senior Director, European Public Policy at Alliance for Regenerative Medicine ATMPs are varied and complex, ranging from potential one-off curative treatments to treatments tailored to a specific individual. As such, they differ significantly from ‘traditional’ medicinal treatments, something which has been recognised by regulators in Europe: the European Parliament introduced ATMPs as a

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HHS Awards Major Funding Award to WFIRM for Lung-on-a-chip Technology Used to Study Toxic Agents and Develop Treatments

Source Wake Forest Baptist Medical Center The Wake Forest Institute for Regenerative Medicine (WFIRM) is the recipient of a major research funding award from the U.S. Department of Health and Human Services (HHS) to support its lung-on-a-chip technology as a model to develop chemical injury treatments. The five-year $24 million program has been approved with an

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The League of European Research Universities shares advice so that patients have a better access to advanced therapy

Source Universitat de Barcelona The League of European Research Universities (LERU) has published a new report on how to improve patients’ access to Advanced Therapy Medicinal Products (ATMPs) in medical centers and university hospitals. In this report, LERU presents recommendations to improve ATMP within the academic context in order to ensure that EU institutions, national

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