Some of the best stuff we picked up around the internet
By: Gorm Palmgren - Mar. 19, 2021
Top pick
To advance the treatment of Duchenne Muscular Dystrophy (DMD), efficient restoration of dystrophin expression in induced myotubes has been achieved using SpCas9 and dual gRNAs. A Danish-Chinese cooperation was behind the achievement, and the results were published in Molecular Therapy - Nucleic Acids.
Research
Long term follow-up of five patients treated with gene therapy (not gene editing) of stem cells for X-linked severe combined immunodeficiency (SCID-X1) shows sustained beneficial effects after 18 years. This happened despite that the body had cleared the corrected stem cells.
Investors wonder if CRISPR Therapeutics can rebound after its stocks lost 12.6% since last month's earnings report. CRISPR Therapeutics reported fourth-quarter 2020 loss per share of $1.50, wider than the Zacks Consensus Estimate of a loss of $1.23.
The Japanese biopharma company AnGes has invested $61 million in Emendo Biotherapeutics to further develop OMNI. It is a pioneering next-generation allele-specific gene-editing platform that uses synthetic biology.
An overview of gene therapies used for the treatment of diseases of the eye is presented in Ophthalmology Times. Larry Hanover wrote the piece that also covers other diseases.
Conferences and events
An online webinar by the International Association of Lawyers will host a webinar on "The Future of Humanity: Genetic Engineering, CRISPR, and Beyond". The webinar will focus on laws and regulations, including those governing medical care, experimentation, intellectual property rights, and the technology commercialisation. You can register for the webinar here.