First CRISPR therapy dosed

  title={First CRISPR therapy dosed},
  journal={Nature Biotechnology},
  • Published 1 April 2020
  • Medicine
  • Nature Biotechnology
16 Citations

Human pluripotent stem cells for the modelling of retinal pigment epithelium homeostasis and disease: A review

The current knowledge gained in understanding the molecular events involved in retinal disease using hPSC‐derived retinal models, in particular RPE models are reviewed.

Early Neural Changes as Underlying Pathophysiological Mechanism in Diabetic Retinopathy

This review will summarize the current status of knowledge regarding pathophysiological mechanisms underlying DR, with a special focus on early neural modifications associated with DR, and describes hyperglycemia-associated molecular and cellular alterations linked to the initiation and progression of DR.

Organelle-level precision with next-generation targeting technologies

Biological pathways that can be exploited to target cargo to specific organelles in vivo are discussed, highlighting nucleic acids as suitable delivery vehicles for organelle-level imaging, diagnosis and therapy.

Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics

The applications of CRISPR-Cas from bench to bedside is summarized and highlights the current obstacles that may limit the usage of CRispr-Cas systems as gene-editing toolkits in precision medicine and offer some viewpoints that may help to tackle these challenges and facilitate technical development.

Therapy Approaches for Stargardt Disease

The most recent pre-clinical and clinical trial data relating to the different strategies being applied to the problem of generating a treatment for the large cohort of Stargardt disease patients worldwide are considered.


  • G. Petris
  • Medicine
    Progress in molecular biology and translational science
  • 2021

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.

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.

Erratic journey of CRISPR/Cas9 in oncology from bench-work to successful-clinical therapy.