First CRISPR therapy dosed

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

Current advances in gene therapy of mitochondrial diseases

Understanding the dynamics of mitochondrial genetics supporting mitochondrial biogenesis is especially important for the development of strategies for the treatment of rare and difficult-to-diagnose diseases.

Gene Editing Technologies to Target HBV cccDNA

The latest developments and applications of gene editing and related approaches for directly targeting HBV DNA and, more specifically, cccDNA in infected hepatocytes are discussed.

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.

Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?

A global survey of researchers working in rare genetic diseases about the future of genetic therapies for the treatment of these diseases over the next 15 years found that in the future genetic therapies will greatly benefit the Treatment of patients with rare Genetic diseases.

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