First test of in-body gene editing shows promise

  title={First test of in-body gene editing shows promise},
  author={Heidi Ledford},
  • Heidi Ledford
  • Published 5 September 2018
  • Medicine, Psychology
  • Nature
Preliminary results suggest that treatment for rare disease is safe, but its effectiveness is unclear. Preliminary results suggest that treatment for rare disease is safe, but its effectiveness is unclear. 

CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases.

Key achievements in gene therapy development and its promising progress with gene editing tools (ZFN, TALEN, CRISPR/Cas9)

A historical overview of gene therapy development is given and recent tools that can be used in precision medicine are described: ZFN, TALEN and CRISPR/Cas9.

Exploiting epigenetics for the treatment of inborn errors of metabolism

An overview of the promises of epigenetics in eventually treating inborn errors of metabolism and the advantages and disadvantages of current and upcoming gene‐based therapies for treatment of IEMs is presented.

Review: Precision Medicine Approaches for Genetic Cardiomyopathy: Targeting Phospholamban R14del

An overview of how precision medicine can be approached as a treatment strategy for genetic heart disease will be discussed and a workflow on how to approach precision medicine in heart disease is proposed.

Trends in Synthetic Biology Applications, Tools, Industry, and Oversight and Their Security Implications.

The trends in synthetic biology applications as well as some of the tools enabling these and future advances are reviewed, and perspective on the security implications is provided, with a focus on US interests.

Schizophrenia: Current Progress and New Treatment Perspective

  • Yuxi WangMinxing Zhang
  • Psychology, Medicine
    2022 International Conference on Biotechnology, Life Science and Medical Engineering (BLSME 2022)
  • 2022
Genetic mutation and environmental factors could both impact on schizophrenia onset and NRG1(Neuregulin 1), a gene closely relates with neural signaling pathway and neural development, is thought to be associated with schizophrenia onset if it is mutated.


  • L. Snyder
  • Biology
    Bacterial Genetics and Genomics
  • 2017
Although most DNA is packaged within the nucleus, mitochondria have a small amount of their own DNA called mitochondrial DNA, and this chapter explores this aspect of genes.

3D engineering for optic neuropathy treatment.