How Genome Editing is Revolutionizing Medicine

Inhaltsverzeichnis (Table of Contents)

• Introduction
• State-of-the-art Gene Editing
  • PAM sequence.
  • Type II CRISPR-Cas System - Cas9 variants and orthologs
    • dCas9
    • eSpCas9
    • SpCas9-HF1
    • HypaCas9
    • xCas9
    • Cas9 Nickases
    • Remarkable SpCas9 Orthologs.
    • Base editing.
    • Prime editing
  • Type V CRISPR-Cas System - Diagnostic tools
    • Cas12a, Cas12b
  • Type VI CRISPR-Cas System - RNA targeting
    • Cas13a.
    • CasRx
  • Transfection methods
    • Non-viral
    • Viral
• Gene Editing and Treatment of Diseases
  • Hemoglobinopathies.
    • CRISPR-correction of sickle cell disease.
    • Reactivation of Fetal Hemoglobin Production
  • Cystic fibrosis
    • Functional repair of the CFTR gene .
    • CFTR repair using Adenine Base Editors
  • Duchenne muscular dystrophy (DMD)
    • CRISPR-Cas9 restores functional dystrophin in mice
    • Long-term evaluation of Dystrophin restoration in mice
  • Treatment of Human Immunodeficiency Virus
    • Genome editing of HIV co-receptors CCR5 and CXCR4.
    • HIV elimination using ART and CRISPR-Cas9
  • Cancer immunotherapy .
    • Editing Primary Human Natural Killer Cells
    • Creating enhanced CAR T Cells using CRISPR-Cas9
    • Creating universal CAR T cells with CRISPR-Cas9
• Gene Editing for Drug Development
  • Glycoengineering
  • Prolonged cultivation
  • Elimination of host cell protein impurities
  • Rapid adaptation cells
  • Enhanced protein production
• Gene Editing Tools for Diagnostics
  • Diagnostic CRISPR endonucleases.
    • Cas12a, Cas12b . . .
    • Advanced endonucleases for diagnostic
    • Cas13a.
  • SHERLOCK.
  • DETECTR
    • SARS-CoV-2 Detection using DETECTR
  • CRISPR targeting of drug-resistant bacteria
    • Targeting pathogenic and drug-resistant bacteria
• Challenges and Obstacles
  • Off-target effects and carcinogenesis
  • Delivery of the CRISPR-Cas system.
  • Immune responses against CRISPR-Cas
• Prospect

Zielsetzung und Themenschwerpunkte (Objectives and Key Themes)

This paper provides an overview of current practices and research areas within the field of modern gene editing, focusing primarily on CRISPR technology. The paper aims to bridge the gap between ongoing advancements in gene editing and public awareness, highlighting its potential and challenges.

• The impact of CRISPR-Cas systems on various aspects of medicine, including the treatment of hereditary diseases, viral infections, and cancer.
• Applications of CRISPR technology in drug development and diagnostics, particularly its use in cell line engineering and rapid pathogen detection.
• A comprehensive overview of the different CRISPR-Cas systems, their variants, and their applications in genome editing and beyond.
• Exploration of the challenges and obstacles associated with clinical applications of CRISPR, such as off-target effects, immune responses, and efficient delivery methods.
• A glimpse into the future of CRISPR technology and its potential to revolutionize medicine and various other fields.

Zusammenfassung der Kapitel (Chapter Summaries)

The paper begins by introducing CRISPR-Cas technology, outlining its basic principles and key components. It then delves into different types of CRISPR-Cas systems, including their variants and applications in various areas such as genome editing, diagnostics, and therapeutics. The paper explores how CRISPR can be used to treat a range of diseases, such as hemoglobinopathies, cystic fibrosis, Duchenne muscular dystrophy, and HIV. Specific examples of CRISPR-based therapies for these diseases are discussed, highlighting the potential of this technology to revolutionize medicine. The paper also examines the use of CRISPR in drug development, particularly in the context of cell line engineering for enhanced protein production and glycoengineering. The potential of CRISPR-based diagnostic tools for rapid pathogen detection and drug-resistant bacteria identification is discussed. Finally, the paper concludes by addressing challenges and obstacles associated with clinical application of CRISPR, including off-target effects, immune responses, and delivery methods. The paper highlights promising developments and future potential of CRISPR technology in various fields.

Schlüsselwörter (Keywords)

CRISPR, gene editing, genome editing, Cas9, Cas12, Cas13, endonucleases, hereditary diseases, viral infections, cancer, drug development, diagnostics, cell line engineering, glycoengineering, off-target effects, immune responses, delivery methods, clinical applications.