Tag Archives: The Stem Cell Blog

Is gene therapy research for single-gene diseases at risk under Trumpcare? – Genetic Literacy Project

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2017-05-26 NEWS

Genetic Literacy Project

Is gene therapy research for single-gene diseases at risk under Trumpcare?
Genetic Literacy Project
The high cost of lifelong frequent infusions or injections of ERT is why the “forever fix” of a gene therapy is an attractive alternative, even if a booster or two becomes necessary. Gene therapy delivers the DNA instructions for making the missing enzyme.

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2017-05-26 NEWS

Genetic Literacy Project

Is gene therapy research for single-gene diseases at risk under Trumpcare?
Genetic Literacy Project
The high cost of lifelong frequent infusions or injections of ERT is why the “forever fix” of a gene therapy is an attractive alternative, even if a booster or two becomes necessary. Gene therapy delivers the DNA instructions for making the missing enzyme.

Continue reading

2017-05-26 NEWS

Medical Xpress

FDA approves cancer drug for personalized immunotherapy approach
Medical Xpress
Experts at the Bloomberg–Kimmel Institute designed the first clinical trial to test the theory that patients whose tumors have defects in mismatch repair genes may respond better to immunotherapy. Results of the trial were presented and published in
SAA Funding Contributes to FDA Approval Of KeytrudaSwimSwam
Expanded Keytruda Approval Signals New Phase for Precision Medicine, May Benefit NGS Panel MakersGenomeWeb

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2017-05-26 NEWS

European Pharmaceutical Review

Abeona Therapeutics receives FDA orphan designation for gene therapy
European Pharmaceutical Review
The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Abeona Therapeutics’s EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa (DEB), including recessive dystrophic epidermolysis …

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2017-05-26 NEWS

Nature.com

Tumour immunology Platelets — a new target in cancer immunotherapy?
Nature.com
Although best recognized for their roles in haemostasis, platelets contribute to other biological processes, including immune regulation. Reporting in Science Immunology, Rachidi et al. show that platelets can suppress T cell responses against tumours …

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2017-05-26 NEWS

Nature.com

Tumour immunology Platelets — a new target in cancer immunotherapy?
Nature.com
Although best recognized for their roles in haemostasis, platelets contribute to other biological processes, including immune regulation. Reporting in Science Immunology, Rachidi et al. show that platelets can suppress T cell responses against tumours …