Description

• Title:
  Applications of gene modification technologies in the treatment of inherited diseases
• Creator:
  Superson, Maciej;
  Szmyt, Katarzyna;
  Wilk-Trytko, Klaudia;
  Krasnoborska, Julia;
  Samojedny, Sylwia;
  Szymańska, Katarzyna;
  Walczak, Kamil
• Description:
  Introduction and Purpose: In last years gene modification technologies such as CRISPR/Cas9 has had a revolutionary impact on the treatment of inherited diseases. Technologies developed from bacterial defense mechanisms, has become a basic tools in scientific research and medical therapies. In our article we provided an overview of applications of gene modifications technologies, directly focusing on CRISPR/Cas9, in genetic disease treatment.   State of Knowledge: New applications of CRISPR/Cas9 are still being explored. Treating inherited diseases such as cystic fibrosis, Duchenne muscular dystrophy, thalassemia, hemophilia, Huntington's disease, Crigler-Najjar syndrome, sickle cell anemia, Marfan syndrome, and phenylketonuria, is feasible with this novel technique. A comparative analysis with other gene editing methods highlights CRISPR/Cas9's efficacy, ease of use, and multiplexing capabilities.  Summary: CRISPR/Cas9 is a groundbreaking technology with broad applications in genetic research and therapy. Its ease of use, cost-effectiveness, and ability to target multiple genes simultaneously position it as a preferred method. However, there are some challenges associated with precision issues and ethical considerations in human embryo gene editing. As CRISPR/Cas9 continues to evolve, responsible application and ethical considerations are important for maximizing its potential in treatment of genetic diseases.
• Object availability:
  free access
• Rights:
  Prawa autorskie (c) 2024 Maciej Superson, Katarzyna Szmyt, Klaudia Wilk-Trytko, Julia Krasnoborska, Sylwia Samojedny, Katarzyna Szymańska, Kamil Walczak;
  https://creativecommons.org/licenses/by-nc-sa/4.0
• Date:
  2024.04.18
• Type:
  info:eu-repo/semantics/article;
  info:eu-repo/semantics/publishedVersion
• Language:
  English
• Source:
  Journal of Education, Health and Sport; Vol. 66 (2024); 50073;
  Journal of Education, Health and Sport; Tom 66 (2024); 50073;
  2391-8306
• Format:
  application/pdf
• Publisher:
  Nicolaus Copernicus University, Toruń, Poland
• Relation: https://apcz.umk.pl/JEHS/article/view/50073/38345
• Subject:
  Crispr/cas9;
  gene modification;
  gene therapy;
  genetic diseases
• Identifier: https://apcz.umk.pl/JEHS/article/view/50073
• Data provider:
  Akademicka Platforma Czasopism
• Can I use it?:
  seek permission
• Type:
  text