Human pluripotent stem-cells (HPSCs)—including human embryonic stem-cells and human induced pluripotent stem-cells—are reshaping the landscape of regenerative medicine.1,2 In Parkinson's disease, ...
Allergy research has historically relied on traditional systems such as 2D cell cultures and mouse models to uncover mechanisms underlying allergic ...
A new CRISPR-based tool that is directly used on patients' cancer cells can identify genes and regulatory elements driving ...
Vector-borne diseases (VBDs) represent a critical challenge in infectious disease control due to their complex transmission dynamics, diverse vector ...
The FDA released a much-anticipated draft guidance on how makers of rare disease genetic medicines can leverage a platform ...
New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for ...
"Gene editing takes centre stage in FDA’s new rare disease approval pathway" was originally created and published by ...
The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would ...
Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more ...
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