GMOs or non-GMOs? The CRISPR Conundrum DOI Creative Commons
Aftab Ahmad, Amer Jamil, Nayla Munawar

et al.

Frontiers in Plant Science, Journal Year: 2023, Volume and Issue: 14

Published: Oct. 9, 2023

CRISPR-Cas9, the “genetic scissors”, is being presaged as a revolutionary technology, having tremendous potential to create designer crops by introducing precise and targeted modifications in genome achieve global food security face of climate change increasing population. Traditional genetic engineering relies on random unpredictable insertion isolated genes or foreign DNA elements into plant genome. However, CRISPR-Cas based gene editing does not necessarily involve inserting element from different species but new traits precisely altering existing genes. CRISPR edited are touching markets, however, world community divided over whether these should be considered genetically modified (GM) non-GM. Classification crops, especially transgene free traditional GM will significantly affect their future public acceptance some regions. Therefore, depending upon regulation non-GMs, perception. Here we briefly discuss how crops. In addition, reagents delivery tools produce transgene-free Moreover, also summarize regulatory classification countries regulating We that controversy CRISPR-edited plants non-GM continue until universal, transparent, scalable framework for introduced worldwide, with increased awareness involving all stakeholders.

Language: Английский

CRISPR/Cas9 therapeutics: progress and prospects DOI Creative Commons
Tianxiang Li, Yanyan Yang, Hongzhao Qi

et al.

Signal Transduction and Targeted Therapy, Journal Year: 2023, Volume and Issue: 8(1)

Published: Jan. 16, 2023

Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety efficient Cas9 variants derivatives have been developed to cope complex genomic changes that occur during diseases. However, strategies effectively deliver CRISPR system diseased cells in vivo are currently lacking, nonviral vectors target recognition functions may be focus research. Pathological physiological resulting from disease onset expected serve as identifying factors targeted delivery targets gene editing. Diseases both varied complex, choice appropriate methods different important. Meanwhile, there still many potential challenges identified when targeting CRISPR/Cas9 treatment. This paper reviews current developments three aspects, namely, type, vector, characteristics. Additionally, this summarizes successful examples clinical trials finally describes possible problems associated applications.

Language: Английский

Citations

296
Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing DOI Creative Commons
Misganaw Asmamaw Mengstie,

Belay Zawdie Wondimu

Biologics, Journal Year: 2021, Volume and Issue: Volume 15, P. 353 - 361

Published: Aug. 1, 2021

Abstract: Clustered regularly interspaced short palindromic repeat (CRISPR) and their associated protein (Cas-9) is the most effective, efficient, accurate method of genome editing tool in all living cells utilized many applied disciplines. Guide RNA (gRNA) CRISPR-associated proteins are two essential components CRISPR/Cas-9 system. The mechanism contains three steps, recognition, cleavage, repair. designed sgRNA recognizes target sequence gene interest through a complementary base pair. While Cas-9 nuclease makes double-stranded breaks at site 3 pair upstream to protospacer adjacent motif, then break repaired by either non-homologous end joining or homology-directed repair cellular mechanisms. genome-editing has wide number applications areas including medicine, agriculture, biotechnology. In it could help design new grains improve nutritional value. being investigated for cancers, HIV, therapy such as sickle cell disease, cystic fibrosis, Duchenne muscular dystrophy. technology also regulation specific genes advanced modification protein. However, immunogenicity, effective delivery systems, off-target effect, ethical issues have been major barriers extend clinical applications. Although becomes era molecular biology countless roles ranging from basic researches applications, there still challenges rub practical various improvements needed overcome obstacles. Keywords: CRISPR, Cas-9, sgRNA, gene-editing, mechanism,

Language: Английский

Citations

273
Fluoropolymers in biomedical applications: state-of-the-art and future perspectives DOI
Jia Lv, Yiyun Cheng

Chemical Society Reviews, Journal Year: 2021, Volume and Issue: 50(9), P. 5435 - 5467

Published: Jan. 1, 2021

Biomedical applications of fluoropolymers in gene delivery, protein drug delivery,19F MRI, PDT, anti-fouling, anti-bacterial, cell culture, and tissue engineering.

Language: Английский

Citations

223
Fluorination Promotes the Cytosolic Delivery of Genes, Proteins, and Peptides DOI
Jia Lv, Hui Wang, Guangyu Rong

et al.

Accounts of Chemical Research, Journal Year: 2022, Volume and Issue: 55(5), P. 722 - 733

Published: Feb. 17, 2022

The cytosolic delivery of biomolecules such as genes, proteins, and peptides is great importance for biotherapy but usually limited by multiple barriers during the process. Cell membrane with high hydrophobic character one representative biological delivery. introduction ligands aliphatic lipids onto materials or could improve their permeability. However, these are lipophilic tend to interact phospholipids in well serum which may hinder efficient intracellular To solve this issue, our research group proposed use fluorous both hydrophobicity lipophobicity ideal alternatives promote delivery.In first attempt, were conjugated cationic polymers increase gene efficacy. fluorination dramatically increased performance at low polymer doses. In addition, strategy greatly improved tolerance polymers, critical vivo. Besides tolerance, mechanism studies revealed that increases steps cellular uptake endosomal escape. Fluorination also allowed assembly low-molecular-weight achieved highly minimal material toxicity. method showed robust efficiency including linear branched dendrimers, bola amphiphilies, dendronized polymers.Besides delivery, fluorinated used protein via a coassembly strategy. For purpose, two lead fluoropolymers screened from library amphiphilic materials. superior nonfluorinated analogues lipids. beneficial encapsulation, reduced denaturation, facilitated endocytosis, decreased toxicity compared exhibited potent efficacy therapeutic peptide achieve cancer therapy able fabricate personalized nanovaccine immunotherapy. Finally, directly disulfide bond Fluorous drive cargo into uniform nanoparticles much proteolytic stability various types cells. traditional techniques cell-penetrating vitro Overall, provide promising strategies biomolecules.

Language: Английский

Citations

101
Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy DOI Creative Commons
Mohammad Chehelgerdi, Matin Chehelgerdi, Milad Khorramian‐Ghahfarokhi

et al.

Molecular Cancer, Journal Year: 2024, Volume and Issue: 23(1)

Published: Jan. 9, 2024

Abstract The CRISPR system is a revolutionary genome editing tool that has the potential to revolutionize field of cancer research and therapy. ability precisely target edit specific genetic mutations drive growth spread tumors opened up new possibilities for development more effective personalized treatments. In this review, we will discuss different CRISPR-based strategies have been proposed therapy, including inactivating genes tumor growth, enhancing immune response cells, repairing cause cancer, delivering cancer-killing molecules directly cells. We also summarize current state preclinical studies clinical trials highlighting most promising results challenges still need be overcome. Safety delivery are important therapy become viable option. limitations overcome, such as off-target effects, safety, site. Finally, provide an overview opportunities in future directions development. change landscape research, review aims overcome realize potential.

Language: Английский

Citations

62
Plant breeding advancements with “CRISPR-Cas” genome editing technologies will assist future food security DOI Creative Commons
Muhammad Ahmad

Frontiers in Plant Science, Journal Year: 2023, Volume and Issue: 14

Published: March 13, 2023

Genome editing techniques are being used to modify plant breeding, which might increase food production sustainably by 2050. A product made feasible genome is becoming better known, because of looser regulation and widespread acceptance. The world’s population supply would never have increased proportionally under current farming practices. development plants has been greatly impacted global warming climate change. Therefore, minimizing these effects crucial for agricultural that sustainable. Crops more resilient abiotic stress sophisticated practices a understanding the response mechanism. Both conventional molecular breeding create viable crop types both processes time-consuming. Recently, breeders shown an interest in approaches genetic manipulation use clustered regularly interspaced short palindromic repeats (CRISPR/Cas9). To ensure security future, kinds with desired traits must be developed. completely new era begun revolution based on CRISPR/CRISPR-associated nuclease (Cas9) systems. All may effectively target particular gene or group loci using Cas9 single-guide RNA (sgRNA). CRISPR/Cas9 can thereby save time labor compared methods. An easy, quick, efficient method directly altering sequences cells CRISPR CRISPR-Cas9 system, was developed from components earliest known bacterial immune allows targeted breakage variety cells/RNA guide endonuclease cleavage specificity system. Editing directed practically any genomic site (gRNA) sequence delivering it cell along endonuclease. We summarize recent research findings, investigate potential applications make predictions about likely future breakthroughs through

Language: Английский

Citations

53
Principles of CRISPR-Cas9 technology: Advancements in genome editing and emerging trends in drug delivery DOI Creative Commons
Alaa A. A. Aljabali, Mohamed El‐Tanani, Murtaza M. Tambuwala

et al.

Journal of Drug Delivery Science and Technology, Journal Year: 2024, Volume and Issue: 92, P. 105338 - 105338

Published: Jan. 6, 2024

The rapid advancement of CRISPR-Cas9 technology has instigated a profound transformation in genome editing with significant implications for fields like health, agriculture, and biotechnology. This abstract provides an overview the historical significance fundamental components CRISPR-Cas9, notably Cas9 protein guide RNA, underscoring its pivotal role genetic manipulation. It emphasizes CRISPR-Cas9's preeminence domain precise editing, driving breakthroughs personalized medicine, gene therapy, agriculture. Of paramount importance is integration nanomaterials, encompassing lipid-based polymeric nanoparticles, alongside viral vectors, serving as potent vehicles augmenting delivery efficiency precision. We explore strategies aimed at enhancing through while also addressing ethical regulatory considerations. In expert opinion section, we offer nuanced perspective on present state field, highlighting potential transformative progress research therapy. stands brink unlocking new possibilities providing innovative solutions to address pressing global challenges.

Language: Английский

Citations

30
Engineering self-deliverable ribonucleoproteins for genome editing in the brain DOI Creative Commons
Kai Chen, Elizabeth C. Stahl, Min Hyung Kang

et al.

Nature Communications, Journal Year: 2024, Volume and Issue: 15(1)

Published: Feb. 26, 2024

The delivery of CRISPR ribonucleoproteins (RNPs) for genome editing in vitro and vivo has important advantages over other methods, including reduced off-target immunogenic effects. However, effective RNPs remains challenging certain cell types due to low efficiency toxicity. To address these issues, we engineer self-deliverable that can promote efficient cellular uptake carry out robust without the need helper materials or biomolecules. Screening cell-penetrating peptides (CPPs) fused CRISPR-Cas9 protein identifies potent constructs capable neural progenitor cells. Further engineering fusion proteins establishes a C-terminal Cas9 with three copies A22p, peptide derived from human semaphorin-3a, exhibits substantially improved efficacy compared constructs. We find generate edits clinically relevant genes when injected directly into mouse striatum. Overall, provide facile platform vivo.

Language: Английский

Citations

18
Advanced Delivery Systems for Gene Editing: A Comprehensive Review from the GenE-HumDi COST Action Working group DOI Creative Commons
Alessia Cavazza, Francisco J Molina-Estevez, Álvaro Plaza Reyes

et al.

Molecular Therapy — Nucleic Acids, Journal Year: 2025, Volume and Issue: 36(1), P. 102457 - 102457

Published: Jan. 18, 2025

Language: Английский

Citations

2
Emerging innate biological properties of nano-drug delivery systems: A focus on PAMAM dendrimers and their clinical potential DOI Creative Commons
Hadeel Kheraldine, Ousama Rachid, Abdella M. Habib

et al.

Advanced Drug Delivery Reviews, Journal Year: 2021, Volume and Issue: 178, P. 113908 - 113908

Published: Aug. 11, 2021

Drug delivery systems or vectors are usually needed to improve the bioavailability and effectiveness of a drug through improving its pharmacokinetics/pharmacodynamics at an organ, tissue cellular level. However, emerging technologies with sensitive readouts as well greater understanding physiological/biological have revealed that polymeric not biologically inert but can innate intrinsic biological actions. In this article, we review multiple biological/toxicological properties naked polyamidoamine (PAMAM) dendrimer in absence any cargo discuss their correlation defined physicochemical PAMAMs terms molecular size (generation), architecture, surface charge chemistry. Further, assess whether reported actions such antimicrobial activity ability sequester glucose modulate key protein interactions cell signaling pathways, be exploited clinically treatment diabetes complications.

Language: Английский

Citations

101