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Delivery of gene editing therapeutics.
Kevadiya, Bhavesh D; Islam, Farhana; Deol, Pallavi; Zaman, Lubaba A; Mosselhy, Dina A; Ashaduzzaman, Md; Bajwa, Neha; Routhu, Nanda Kishore; Singh, Preet Amol; Dawre, Shilpa; Vora, Lalitkumar K; Nahid, Sumaiya; Mathur, Deepali; Nayan, Mohammad Ullah; Baldi, Ashish; Kothari, Ramesh; Patel, Tapan A; Madan, Jitender; Gounani, Zahra; Bariwal, Jitender; Hettie, Kenneth S; Gendelman, Howard E.
Afiliação
  • 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.
  • 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
  • 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: p
  • 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.
  • 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 Ins
  • Ashaduzzaman M; Department of Computer Science, University of Nebraska Omaha, Omaha, NE 68182, USA. Electronic address: aashaduzzaman@unomaha.edu.
  • Bajwa N; University Institute of Pharma Sciences, Chandigarh University, Mohali, Punjab, India. Electronic address: nehabajwa2765@gmail.com.
  • 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.
  • 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.
  • 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.
  • 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.
  • Nahid S; Department of Pharmaceutical Sciences, University of Nebraska Medical Center, Omaha, NE 68198-5880, USA. Electronic address: snahid@unmc.edu.
  • Mathur D; Neuro Care Centre, Bhubaneswar, Odisha 751022, India. Electronic address: matdeepali@gmail.com.
  • 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.
  • 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.
  • Kothari R; Department of Biosciences, Saurashtra University, Rajkot 360005, Gujarat, India. Electronic address: kothari1971@gmail.com.
  • Patel TA; Department of Cellular and Integrative Physiology, University of Nebraska Medical Center, Omaha, NE 68198, USA. Electronic address: tapatel84@gmail.com.
  • Madan J; Department of Pharmaceutics, National Institute of Pharmaceutical Education and Research-NIPER, Hyderabad 500037, Telangana, India. Electronic address: jitenderpharmacy@gmail.com.
  • Gounani Z; Division of Pharmaceutical Biosciences, Faculty of Pharmacy, University of Helsinki, Viikinkaari 5, 00790 Helsinki, Finland. Electronic address: zahra.gounani@helsinki.fi.
  • 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.
  • 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.
  • 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@un
Nanomedicine ; 54: 102711, 2023 Nov.
Article em En | MEDLINE | ID: mdl-37813236
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Sistemas CRISPR-Cas / Edição de Genes Idioma: En Revista: Nanomedicine Assunto da revista: BIOTECNOLOGIA Ano de publicação: 2023 Tipo de documento: Article País de publicação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Sistemas CRISPR-Cas / Edição de Genes Idioma: En Revista: Nanomedicine Assunto da revista: BIOTECNOLOGIA Ano de publicação: 2023 Tipo de documento: Article País de publicação: Estados Unidos