CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective

Advanced Science, Journal Year: 2023, Volume and Issue: 10(19)

Published: May 11, 2023

Clustered randomly interspaced short palindromic repeats (CRISPRs) and its associated endonuclease protein, i.e., Cas9, have been discovered as an immune system in bacteria archaea; nevertheless, they are now being adopted mainstream biotechnological/molecular scissors that can modulate ample genetic nongenetic diseases via insertion/deletion, epigenome editing, messenger RNA CRISPR interference, etc. Many Food Drug Administration-approved ongoing clinical trials on adopt ex vivo strategies, wherein the gene editing is performed vivo, followed by reimplantation to patients. However, delivery of components still under preclinical surveillance. This review has summarized nonviral nanodelivery strategies for using CRISPR/Cas9 recent advancements, strategic points view, challenges, future aspects tissue-specific nanomaterials.

Language: Английский

---

Toward the Development of Epigenome Editing-Based Therapeutics: Potentials and Challenges

International Journal of Molecular Sciences, Journal Year: 2023, Volume and Issue: 24(5), P. 4778 - 4778

Published: March 1, 2023

The advancement in epigenetics research over the past several decades has led to potential application of epigenome-editing technologies for treatment various diseases. In particular, epigenome editing is potentially useful genetic and other related diseases, including rare imprinted as it can regulate expression target region, thereby causative gene, with minimal or no modification genomic DNA. Various efforts are underway successfully apply vivo, such improving specificity, enzymatic activity, drug delivery development reliable therapeutics. this review, we introduce latest findings, summarize current limitations future challenges practical disease therapy, important factors consider, chromatin plasticity, a more effective editing-based therapy.

Language: Английский

---

The CRISPR/Cas9 System Delivered by Extracellular Vesicles

Pharmaceutics, Journal Year: 2023, Volume and Issue: 15(3), P. 984 - 984

Published: March 18, 2023

Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein (Cas) systems can precisely manipulate DNA sequences to change the characteristics of cells and organs, which has potential in mechanistic research on genes treatment diseases. However, clinical applications are restricted by lack safe, targeted effective delivery vectors. Extracellular vesicles (EVs) an attractive platform for CRISPR/Cas9. Compared with viral other vectors, EVs present several advantages, including safety, protection, capacity, penetrating ability, targeting ability modification. Consequently, profitably used deliver CRISPR/Cas9 vivo. In this review, advantages disadvantages form vectors concluded. The favorable traits as such innate characteristics, physiological pathological functions, safety EVs, summarized. Furthermore, terms EV sources isolation strategies, loading methods have been concluded discussed. Finally, review provides future directions system applications, consistent quality, yield EVs.

Language: Английский

---

Engineered Nanomaterials to Potentiate CRISPR/Cas9 Gene Editing for Cancer Therapy

Advanced Materials, Journal Year: 2023, Volume and Issue: 36(13)

Published: July 12, 2023

Abstract Clustered regularly interspaced short palindromic repeats/associated protein 9 (CRISPR/Cas9) gene‐editing technology shows promise for manipulating single or multiple tumor‐associated genes and engineering immune cells to treat cancers. Currently, most strategies rely on viral delivery; yet, while being efficient, many limitations, mainly from safety packaging capacity considerations, hinder the use of CRISPR vectors in cancer therapy. In contrast, recent advances non‐viral CRISPR/Cas9 nanoformulations have paved way better gene editing, as these can be engineered improve safety, efficiency, specificity through optimizing capacity, pharmacokinetics, targetability. this review, advance delivery is highlighted, there a discussion how approaches potentially used cancers addressing aforementioned followed by perspectives designing proper CRISPR/Cas9‐based nanomedicine system with translational potential.

Language: Английский

---

Rational design and combinatorial chemistry of ionizable lipids for RNA delivery

Journal of Materials Chemistry B, Journal Year: 2023, Volume and Issue: 11(28), P. 6527 - 6539

Published: Jan. 1, 2023

This review will delve into the crucial role of ionizable lipids in development lipid nanoparticles (LNPs) for efficient RNA delivery.

Language: Английский

---