A new era of gene editing for the treatment of human diseases

Mandip Kc, J. Steer Clifford

Research output: Contribution to journalReview article

Abstract

Summary The treatment of human diseases using gene-editing technology has been envisioned for several decades with the realisation that so many were associated with mutations in DNA. The Human Genome Project opened new doors for identifying the genetic bases for human suffering. Research on gene editing has been active since the 1970s, but the technology has seen substantial growth and application just within the past decade. Simply stated, CRISPR technology has become a phenomenon in both biomedical and therapeutics research. Concurrently, cell therapies and pluripotent stem cell research have also been refined and now interfaced with CRISPR technology to enhance and maximise their potential in modelling as well as treatment of human diseases. In this review, we discuss the novel and revolutionary modality of gene editing, as this marks a new era in research and medicine. We also discuss gene-modifying technologies leading to CRISPR, as they are still being used for a wide variety of genomic applications. The modes and challenges for delivery of gene editing components are also discussed. Lastly, we review examples of human diseases that are not only amenable to gene editing techniques, but also show true promise of cure in the early 21st century of genetic correction and gene repair.

Original languageEnglish (US)
Article numberw20021
JournalSwiss Medical Weekly
Volume149
Issue number3-4
DOIs
StatePublished - Jan 1 2019

Fingerprint

Clustered Regularly Interspaced Short Palindromic Repeats
Technology
Therapeutic Human Experimentation
Gene Components
Human Genome Project
Therapeutics
Stem Cell Research
Pluripotent Stem Cells
Medical Genetics
Cell- and Tissue-Based Therapy
Psychological Stress
Research
Genes
Biomedical Research
Medicine
Gene Editing
Mutation
DNA
Growth

Keywords

  • CRISPR
  • gene editing
  • human genetic diseases

PubMed: MeSH publication types

  • Journal Article
  • Review

Cite this

A new era of gene editing for the treatment of human diseases. / Kc, Mandip; Steer Clifford, J.

In: Swiss Medical Weekly, Vol. 149, No. 3-4, w20021, 01.01.2019.

Research output: Contribution to journalReview article

@article{3c581c40a1c64167802d4218b3b6cc3b,
title = "A new era of gene editing for the treatment of human diseases",
abstract = "Summary The treatment of human diseases using gene-editing technology has been envisioned for several decades with the realisation that so many were associated with mutations in DNA. The Human Genome Project opened new doors for identifying the genetic bases for human suffering. Research on gene editing has been active since the 1970s, but the technology has seen substantial growth and application just within the past decade. Simply stated, CRISPR technology has become a phenomenon in both biomedical and therapeutics research. Concurrently, cell therapies and pluripotent stem cell research have also been refined and now interfaced with CRISPR technology to enhance and maximise their potential in modelling as well as treatment of human diseases. In this review, we discuss the novel and revolutionary modality of gene editing, as this marks a new era in research and medicine. We also discuss gene-modifying technologies leading to CRISPR, as they are still being used for a wide variety of genomic applications. The modes and challenges for delivery of gene editing components are also discussed. Lastly, we review examples of human diseases that are not only amenable to gene editing techniques, but also show true promise of cure in the early 21st century of genetic correction and gene repair.",
keywords = "CRISPR, gene editing, human genetic diseases",
author = "Mandip Kc and {Steer Clifford}, J.",
year = "2019",
month = "1",
day = "1",
doi = "10.4414/smw.2019.20021",
language = "English (US)",
volume = "149",
journal = "Swiss Medical Weekly",
issn = "1424-7860",
publisher = "EMH Swiss Medical Publishers Ltd.",
number = "3-4",

}

TY - JOUR

T1 - A new era of gene editing for the treatment of human diseases

AU - Kc, Mandip

AU - Steer Clifford, J.

PY - 2019/1/1

Y1 - 2019/1/1

N2 - Summary The treatment of human diseases using gene-editing technology has been envisioned for several decades with the realisation that so many were associated with mutations in DNA. The Human Genome Project opened new doors for identifying the genetic bases for human suffering. Research on gene editing has been active since the 1970s, but the technology has seen substantial growth and application just within the past decade. Simply stated, CRISPR technology has become a phenomenon in both biomedical and therapeutics research. Concurrently, cell therapies and pluripotent stem cell research have also been refined and now interfaced with CRISPR technology to enhance and maximise their potential in modelling as well as treatment of human diseases. In this review, we discuss the novel and revolutionary modality of gene editing, as this marks a new era in research and medicine. We also discuss gene-modifying technologies leading to CRISPR, as they are still being used for a wide variety of genomic applications. The modes and challenges for delivery of gene editing components are also discussed. Lastly, we review examples of human diseases that are not only amenable to gene editing techniques, but also show true promise of cure in the early 21st century of genetic correction and gene repair.

AB - Summary The treatment of human diseases using gene-editing technology has been envisioned for several decades with the realisation that so many were associated with mutations in DNA. The Human Genome Project opened new doors for identifying the genetic bases for human suffering. Research on gene editing has been active since the 1970s, but the technology has seen substantial growth and application just within the past decade. Simply stated, CRISPR technology has become a phenomenon in both biomedical and therapeutics research. Concurrently, cell therapies and pluripotent stem cell research have also been refined and now interfaced with CRISPR technology to enhance and maximise their potential in modelling as well as treatment of human diseases. In this review, we discuss the novel and revolutionary modality of gene editing, as this marks a new era in research and medicine. We also discuss gene-modifying technologies leading to CRISPR, as they are still being used for a wide variety of genomic applications. The modes and challenges for delivery of gene editing components are also discussed. Lastly, we review examples of human diseases that are not only amenable to gene editing techniques, but also show true promise of cure in the early 21st century of genetic correction and gene repair.

KW - CRISPR

KW - gene editing

KW - human genetic diseases

UR - http://www.scopus.com/inward/record.url?scp=85060602725&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85060602725&partnerID=8YFLogxK

U2 - 10.4414/smw.2019.20021

DO - 10.4414/smw.2019.20021

M3 - Review article

VL - 149

JO - Swiss Medical Weekly

JF - Swiss Medical Weekly

SN - 1424-7860

IS - 3-4

M1 - w20021

ER -