First CRISPR therapy dosed

  title={First CRISPR therapy dosed},
  journal={Nature Biotechnology},
  • Published 2020
  • Medicine
  • Nature Biotechnology
12 Citations
Advancing Drug Discovery for Neurological Disorders Using iPSC-Derived Neural Organoids
Though several open issues still hamper the full implementation of the organoid technology outside academia, rapid progress in this field will help to prompt its translation toward large-scale drug screening for neurological disorders. Expand
Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics
  • Wenyi Liu, Luoxi Li, Jianxin Jiang, Min Wu, Ping Lin
  • Precision Clinical Medicine
  • 2021
Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)-associated systems (Cas) are efficient tools for targeting specific genes for laboratory research, agriculturalExpand
CRISPR-based strategies in infectious disease diagnosis and therapy
CRISPR technologies can be harnessed to create rapid, low-cost diagnostic systems, as well as to identify drug-resistance genes and offer solutions to many of the most challenging human infections. Expand
Erratic journey of CRISPR/Cas9 in oncology from bench-work to successful-clinical therapy.
This review explores the literature around the mechanism of Nobel winning CRISPR/Cas9 and its journey from its discovery to various pre-clinical and clinical trials in oncology, focusing mostly on PD-1 knockout CAR-T cell therapy. Expand
From Antisense RNA to RNA Modification: Therapeutic Potential of RNA-Based Technologies
The current FDA-approved antisense MoA and three novel modalities based on post-transcriptional RNA modifications with therapeutic potential, including ADAR (Adenosine deaminases acting on RNA)-mediated RNA editing, targeted pseudouridylation, and 2′-O-methylation are reviewed. Expand
Long-term Stable Reduction of Low-density Lipoprotein in Nonhuman Primates Following In Vivo Genome Editing of PCSK9.
It is demonstrated that targeted in vivo gene disruption exerts a lasting therapeutic effect and provide pivotal data for safety considerations which support clinical translation. Expand
  • G. Petris
  • Medicine
  • Progress in molecular biology and translational science
  • 2021
The growing complexity of COVID-19 drug and vaccine candidates: challenges and critical transitions
This review aims to provide an updated insight of the current research, development and trials on repurposing existing drugs and preventive intervention for COVID-19, along with the related issues, complexities and challenges, especially after the observed high transmissibility lately. Expand
Therapy Approaches for Stargardt Disease
Despite being the most prevalent cause of inherited blindness in children, Stargardt disease is yet to achieve the same clinical trial success as has been achieved for other inherited retinalExpand
Focused Update on AAV-Based Gene Therapy Clinical Trials for Inherited Retinal Degeneration.
Pre-clinical progress in AAV-based therapies aimed at treating other ocular conditions that can have hereditable links, and what alternative technologies are progressing in the same therapeutic space are discussed. Expand