Delivery of gene editing therapeutics.

Gene Editing CRISPR-Cas Systems / genetics CRISPR-Associated Protein 9 / genetics Gene Transfer Techniques Genetic Therapy
CRISPR-Cas9 Gene editing Lipid nanoparticles Viral vector delivery

Journal

Nanomedicine : nanotechnology, biology, and medicine
ISSN: 1549-9642
Titre abrégé: Nanomedicine
Pays: United States
ID NLM: 101233142

Informations de publication

Date de publication:
Nov 2023
Historique:
received: 24 04 2023
revised: 31 08 2023
accepted: 15 09 2023
medline: 6 11 2023
pubmed: 10 10 2023
entrez: 9 10 2023
Statut: ppublish

Résumé

For the past decades, gene editing demonstrated the potential to attenuate each of the root causes of genetic, infectious, immune, cancerous, and degenerative disorders. More recently, Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 (CRISPR-Cas9) editing proved effective for editing genomic, cancerous, or microbial DNA to limit disease onset or spread. However, the strategies to deliver CRISPR-Cas9 cargos and elicit protective immune responses requires safe delivery to disease targeted cells and tissues. While viral vector-based systems and viral particles demonstrate high efficiency and stable transgene expression, each are limited in their packaging capacities and secondary untoward immune responses. In contrast, the nonviral vector lipid nanoparticles were successfully used for as vaccine and therapeutic deliverables. Herein, we highlight each available gene delivery systems for treating and preventing a broad range of infectious, inflammatory, genetic, and degenerative diseases. STATEMENT OF SIGNIFICANCE: CRISPR-Cas9 gene editing for disease treatment and prevention is an emerging field that can change the outcome of many chronic debilitating disorders.

Identifiants

DOI: 10.1016/j.nano.2023.102711 PMID: 37813236
pubmed: 37813236
pii: S1549-9634(23)00062-X
doi: 10.1016/j.nano.2023.102711
pii:
doi:

Substances chimiques

CRISPR-Associated Protein 9 EC 3.1.-

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

102711

Subventions

Organisme : NIAID NIH HHS
ID : R01 AI145542
Pays : United States
Organisme : NIAID NIH HHS
ID : R01 AI158160
Pays : United States
Organisme : NIMH NIH HHS
ID : R01 MH121402
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS034239
Pays : United States

Informations de copyright

Copyright © 2023 Elsevier Inc. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of competing interest The authors have no known competing financial interests or personal relationships that would have influence what is reported in this paper.

Auteurs

Bhavesh D Kevadiya (BD)

Department of Pharmacology and Experimental Neuroscience, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA. Electronic address: bbhaveshpatel@gmail.com.

Farhana Islam (F)

Department of Pharmacology and Experimental Neuroscience, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA; Department of Biochemistry and Molecular Biology, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA. Electronic address: farhana.islam@unmc.edu.

Pallavi Deol (P)

Department of Pharmacology and Experimental Neuroscience, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA; Institute of Modeling Collaboration and Innovation and Department of Biological Sciences, University of Idaho, Moscow, ID 83844, USA. Electronic address: pallavi.deol@gmail.com.

Lubaba A Zaman (LA)

Department of Pharmacology and Experimental Neuroscience, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA. Electronic address: lubaba.zaman@unmc.edu.

Dina A Mosselhy (DA)

Department of Virology, Faculty of Medicine, University of Helsinki, P.O. Box 21, 00014 Helsinki, Finland; Department of Veterinary Biosciences, Faculty of Veterinary Medicine, University of Helsinki, 00014 Helsinki, Finland; Microbiological Unit, Fish Diseases Department, Animal Health Research Institute, ARC, Dokki, Giza 12618, Egypt. Electronic address: dina_mosselhy@hotmail.com.

Md Ashaduzzaman (M)

Department of Computer Science, University of Nebraska Omaha, Omaha, NE 68182, USA. Electronic address: aashaduzzaman@unomaha.edu.

Neha Bajwa (N)

University Institute of Pharma Sciences, Chandigarh University, Mohali, Punjab, India. Electronic address: nehabajwa2765@gmail.com.

Nanda Kishore Routhu (NK)

Emory Vaccine Center, Emory National Primate Research Center, Emory University, Atlanta, GA 30329, USA; Department of Pathology and Laboratory Medicine, Emory University School of Medicine, Atlanta, GA, USA. Electronic address: nkrouthu@gmail.com.

Preet Amol Singh (PA)

University Institute of Pharma Sciences, Chandigarh University, Mohali, Punjab, India; Department of Pharmaceutical Sciences and Technology, Maharaja Ranjit Singh Punjab Technical University, Bathinda, Punjab. Electronic address: preetnabha67@gmail.com.

Shilpa Dawre (S)

Department of Pharmaceutics, School of Pharmacy & Technology Management, SVKMs, NMIMS, Babulde Banks of Tapi River, MPTP Park, Mumbai-Agra Road, Shirpur, Maharashtra, 425405, India. Electronic address: shilpadawre@gmail.com.

Lalitkumar K Vora (LK)

School of Pharmacy, Queen's University Belfast, Medical Biology Centre, 97 Lisburn Road, Belfast BT9 7BL, United Kingdom. Electronic address: L.vora@qub.ac.uk.

Sumaiya Nahid (S)

Department of Pharmaceutical Sciences, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA. Electronic address: snahid@unmc.edu.

Deepali Mathur (D)

Neuro Care Centre, Bhubaneswar, Odisha 751022, India. Electronic address: matdeepali@gmail.com.

Mohammad Ullah Nayan (MU)

Department of Biochemistry and Molecular Biology, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA. Electronic address: mohammadullah.nayan@unmc.edu.

Ashish Baldi (A)

University Institute of Pharma Sciences, Chandigarh University, Mohali, Punjab, India; Department of Pharmaceutical Sciences and Technology, Maharaja Ranjit Singh Punjab Technical University, Bathinda, Punjab. Electronic address: baldiashish@gmail.com.

Ramesh Kothari (R)

Department of Biosciences, Saurashtra University, Rajkot 360005, Gujarat, India. Electronic address: kothari1971@gmail.com.

Tapan A Patel (TA)

Department of Cellular and Integrative Physiology, University of Nebraska Medical Center, Omaha, NE 68198, USA. Electronic address: tapatel84@gmail.com.

Jitender Madan (J)

Department of Pharmaceutics, National Institute of Pharmaceutical Education and Research-NIPER, Hyderabad 500037, Telangana, India. Electronic address: jitenderpharmacy@gmail.com.

Zahra Gounani (Z)

Division of Pharmaceutical Biosciences, Faculty of Pharmacy, University of Helsinki, Viikinkaari 5, 00790 Helsinki, Finland. Electronic address: zahra.gounani@helsinki.fi.

Jitender Bariwal (J)

Department of Cell Physiology and Molecular Biophysics, Center for Membrane Protein Research, Texas Tech University Health Sciences Center, School of Medicine, 3601 4th Street, Lubbock, TX 79430-6551, USA. Electronic address: jitender.bariwal@gmail.com.

Kenneth S Hettie (KS)

Molecular Imaging Program at Stanford (MIPS), Department of Radiology, Department of Otolaryngology - Head & Neck Surgery, Stanford University School of Medicine, Stanford, CA 94305, USA. Electronic address: khettie@stanford.edu.

Howard E Gendelman (HE)

Department of Pharmacology and Experimental Neuroscience, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA; Department of Pathology and Microbiology, College of Medicine, University of Nebraska Medical Center, Omaha, NE 68198, USA. Electronic address: hegendel@unmc.edu.

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Classifications MeSH

questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Édition de gène › Delivery of gene editing therapeutics.
questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › Épigenèse génétique › Extinction de l'expression des gènes › Systèmes CRISPR-Cas › Delivery of gene editing therapeutics.
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines associées aux CRISPR › Protéine-9 associée à CRISPR › Delivery of gene editing therapeutics.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Techniques de transfert de gènes › Delivery of gene editing therapeutics.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Delivery of gene editing therapeutics.