CRISPR/CAS9 And Gene Editing in Drug Delivery: Emerging Delivery Systems for Gene Therapy and Genome

GENETIC EDITING: CRISPR/Cas9 technologies have revolutionised genome editing. They offer unmatched precision, speed, and versatility. Familiar with the immune system of bacteria, it is now a powerful tool. It treats genetic diseases, infectious diseases, and cancer. And even as an omen, its clinical effectiveness demands reliable, precise means of administration. These systems have created viral and non-viral platforms. The list includes: lipid nanoparticles (LNPs), viral vectors (AAVs), and polymeric nanoparticles. New delivery technologies, like SORT LNPs and VLPs, have improved targeting. They offer better therapy and fewer off-target effects.Nonetheless, significant obstacles remain. Delivery efficiencies, immunogenicity and scalability are huge hurdles to wide-scale clinical deployment. To reduce these difficulties, we can develop small Cas variants like Cas12a. We can also engineer guide RNAs (gRNAs) for better precision and fewer off-target effects. There are promising prospects. They come from AI-optimised, hybrid delivery platforms. They are virally-free and non-viral. The FDA's approval of CRISPR drugs for sickle cell disease shows their potential to cure some incurable diseases. Trials now include rare genetic diseases, cancers and metabolic disorders. No matter how big it is, production scale and cost are a bottleneck. We still need better manufacturing and quality control practices. This review examines the latest advances in CRISPR/Cas9 delivery methods. It covers the barriers to clinical use and the future of this groundbreaking technology. CRISPR/Cas9 could revolutionise medicine. It may provide treatments that target the root causes of diseases. It could overcome current limits and use new discoveries.

Authors

Publication Details

Journal
International Journal of Drug Delivery Technology
Published
2025-03-25
DOI
https://doi.org/10.25258/ijddt.15.1.46
Citations
1
Primary Topic
CRISPR and Genetic Engineering
Type
article
Field-Weighted Citation Impact
0.81
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article

CRISPR/CAS9 And Gene Editing in Drug Delivery: Emerging Delivery Systems for Gene Therapy and Genome

Sushil Kumar Varma, Gayatri Patil
1 citations
International Journal of Drug Delivery Technology
CRISPR and Genetic Engineering
0.81
article

CRISPR/CAS9 And Gene Editing in Drug Delivery: Emerging Delivery Systems for Gene Therapy and Genome

Sushil Kumar Varma, Gayatri Patil
article en
1 citations

Abstract

GENETIC EDITING: CRISPR/Cas9 technologies have revolutionised genome editing. They offer unmatched precision, speed, and versatility. Familiar with the immune system of bacteria, it is now a powerful tool. It treats genetic diseases, infectious diseases, and cancer. And even as an omen, its clinical effectiveness demands reliable, precise means of administration. These systems have created viral and non-viral platforms. The list includes: lipid nanoparticles (LNPs), viral vectors (AAVs), and polymeric nanoparticles. New delivery technologies, like SORT LNPs and VLPs, have improved targeting. They offer better therapy and fewer off-target effects.Nonetheless, significant obstacles remain. Delivery efficiencies, immunogenicity and scalability are huge hurdles to wide-scale clinical deployment. To reduce these difficulties, we can develop small Cas variants like Cas12a. We can also engineer guide RNAs (gRNAs) for better precision and fewer off-target effects. There are promising prospects. They come from AI-optimised, hybrid delivery platforms. They are virally-free and non-viral. The FDA's approval of CRISPR drugs for sickle cell disease shows their potential to cure some incurable diseases. Trials now include rare genetic diseases, cancers and metabolic disorders. No matter how big it is, production scale and cost are a bottleneck. We still need better manufacturing and quality control practices. This review examines the latest advances in CRISPR/Cas9 delivery methods. It covers the barriers to clinical use and the future of this groundbreaking technology. CRISPR/Cas9 could revolutionise medicine. It may provide treatments that target the root causes of diseases. It could overcome current limits and use new discoveries.

International Journal of Drug Delivery TechnologyVol. 15(01)
Openalex Percentile: Top 29%
CRISPR and Genetic Engineering
0.81
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