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Article ; Online: Delivery Approaches for Therapeutic Genome Editing and Challenges.

Ates, Ilayda / Rathbone, Tanner / Stuart, Callie / Bridges, P Hudson / Cottle, Renee N

2020 Volume 11, Issue 10

Abstract: ... for local and systemic delivery of gene-editing reagents. In this review, we elaborate on the challenges and ... clinical trials, a major barrier for therapeutic gene editing is the absence of safe and effective methods ... Impressive therapeutic advances have been possible through the advent of zinc-finger nucleases and ...

Abstract	Impressive therapeutic advances have been possible through the advent of zinc-finger nucleases and transcription activator-like effector nucleases. However, discovery of the more efficient and highly tailorable clustered regularly interspaced short palindromic repeats (CRISPR) and associated proteins (Cas9) has provided unprecedented gene-editing capabilities for treatment of various inherited and acquired diseases. Despite recent clinical trials, a major barrier for therapeutic gene editing is the absence of safe and effective methods for local and systemic delivery of gene-editing reagents. In this review, we elaborate on the challenges and provide practical considerations for improving gene editing. Specifically, we highlight issues associated with delivery of gene-editing tools into clinically relevant cells.
MeSH term(s)	CRISPR-Cas Systems ; Gene Editing ; Gene Transfer Techniques/standards ; Genetic Therapy ; Genetic Vectors/administration & dosage ; Humans
Keywords	covid19
Language	English
Publishing date	2020-09-23
Publishing country	Switzerland
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't ; Review
ZDB-ID	2527218-4
ISSN	2073-4425 ; 2073-4425
ISSN (online)	2073-4425
ISSN	2073-4425
DOI	10.3390/genes11101113
Database	MEDical Literature Analysis and Retrieval System OnLINE

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Article: Delivery Approaches for Therapeutic Genome Editing and Challenges

Ates, Ilayda / Rathbone, Tanner / Stuart, Callie / Bridges, P. Hudson / Cottle, Renee N

Genes. 2020 Sept. 23, v. 11, no. 10

2020

Abstract	Impressive therapeutic advances have been possible through the advent of zinc-finger nucleases and transcription activator-like effector nucleases. However, discovery of the more efficient and highly tailorable clustered regularly interspaced short palindromic repeats (CRISPR) and associated proteins (Cas9) has provided unprecedented gene-editing capabilities for treatment of various inherited and acquired diseases. Despite recent clinical trials, a major barrier for therapeutic gene editing is the absence of safe and effective methods for local and systemic delivery of gene-editing reagents. In this review, we elaborate on the challenges and provide practical considerations for improving gene editing. Specifically, we highlight issues associated with delivery of gene-editing tools into clinically relevant cells.
Keywords	clinical trials ; gene editing ; nucleases ; proteins ; therapeutics ; zinc finger motif
Language	English
Dates of publication	2020-0923
Publishing place	Multidisciplinary Digital Publishing Institute
Document type	Article
ZDB-ID	2527218-4
ISSN	2073-4425
ISSN	2073-4425
DOI	10.3390/genes11101113
Database	NAL-Catalogue (AGRICOLA)

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Article ; Online: Delivery platforms for broadly neutralizing antibodies.

Joshi, Lok R / Gálvez, Nicolás M S / Ghosh, Sukanya / Weiner, David B / Balazs, Alejandro B

Current opinion in HIV and AIDS

2023 Volume 18, Issue 4, Page(s) 191–208

Abstract: ... evaluated as a therapeutic approach to prevent or treat HIV infections. However, a number of challenges face ... Although each approach has unique strengths and weaknesses, additional advances in efficiency of gene delivery and ... of bNAbs, gene-based delivery approaches have been developed which overcome the limitations of passive ...

Abstract	Purpose of review: Passive administration of broadly neutralizing antibodies (bNAbs) is being evaluated as a therapeutic approach to prevent or treat HIV infections. However, a number of challenges face the widespread implementation of passive transfer for HIV. To reduce the need of recurrent administrations of bNAbs, gene-based delivery approaches have been developed which overcome the limitations of passive transfer. Recent findings: The use of DNA and mRNA for the delivery of bNAbs has made significant progress. DNA-encoded monoclonal antibodies (DMAbs) have shown great promise in animal models of disease and the underlying DNA-based technology is now being tested in vaccine trials for a variety of indications. The COVID-19 pandemic greatly accelerated the development of mRNA-based technology to induce protective immunity. These advances are now being successfully applied to the delivery of monoclonal antibodies using mRNA in animal models. Delivery of bNAbs using viral vectors, primarily adeno-associated virus (AAV), has shown great promise in preclinical animal models and more recently in human studies. Most recently, advances in genome editing techniques have led to engineering of monoclonal antibody expression from B cells. These efforts aim to turn B cells into a source of evolving antibodies that can improve through repeated exposure to the respective antigen. Summary: The use of these different platforms for antibody delivery has been demonstrated across a wide range of animal models and disease indications, including HIV. Although each approach has unique strengths and weaknesses, additional advances in efficiency of gene delivery and reduced immunogenicity will be necessary to drive widespread implementation of these technologies. Considering the mounting clinical evidence of the potential of bNAbs for HIV treatment and prevention, overcoming the remaining technical challenges for gene-based bNAb delivery represents a relatively straightforward path towards practical interventions against HIV infection.
MeSH term(s)	Animals ; Humans ; HIV Infections/prevention & control ; Broadly Neutralizing Antibodies ; HIV Antibodies ; Antibodies, Neutralizing ; Pandemics ; HIV-1/genetics ; COVID-19/therapy ; Antibodies, Monoclonal/genetics
Chemical Substances	Broadly Neutralizing Antibodies ; HIV Antibodies ; Antibodies, Neutralizing ; Antibodies, Monoclonal
Language	English
Publishing date	2023-05-15
Publishing country	United States
Document type	Review ; Journal Article ; Research Support, N.I.H., Extramural
ZDB-ID	2502511-9
ISSN	1746-6318 ; 1746-630X
ISSN (online)	1746-6318
ISSN	1746-630X
DOI	10.1097/COH.0000000000000803
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Article ; Online: Cellular Trafficking of Nanotechnology-Mediated mRNA Delivery.

Huang, Pei / Deng, Hongzhang / Wang, Changrong / Zhou, Yongfeng / Chen, Xiaoyuan

Advanced materials (Deerfield Beach, Fla.)

2023 Volume 36, Issue 13, Page(s) e2307822

Abstract: ... therapeutic approach that involves technologies for both mRNA itself and the delivery vehicle. Although there ... introduced, including immunotherapy, protein replacement therapy, genome editing, and cellular reprogramming ... and translation of mRNA into protein. Finally, the challenges and opportunities for the development ...

Abstract	Messenger RNA (mRNA)-based therapy has emerged as a powerful, safe, and rapidly scalable therapeutic approach that involves technologies for both mRNA itself and the delivery vehicle. Although there are some unique challenges for different applications of mRNA therapy, a common challenge for all mRNA therapeutics is the transport of mRNA into the target cell cytoplasm for sufficient protein expression. This review is focused on the behaviors at the cellular level of nanotechnology-mediated mRNA delivery systems, which have not been comprehensively reviewed yet. First, the four main therapeutic applications of mRNA are introduced, including immunotherapy, protein replacement therapy, genome editing, and cellular reprogramming. Second, common types of mRNA cargos and mRNA delivery systems are summarized. Third, strategies to enhance mRNA delivery efficiency during the cellular trafficking process are highlighted, including accumulation to the cell, internalization into the cell, endosomal escape, release of mRNA from the nanocarrier, and translation of mRNA into protein. Finally, the challenges and opportunities for the development of nanotechnology-mediated mRNA delivery systems are presented. This review can provide new insights into the future fabrication of mRNA nanocarriers with desirable cellular trafficking performance.
MeSH term(s)	RNA, Messenger/metabolism ; Nanoparticles/metabolism ; Nanotechnology ; Endosomes/metabolism ; Proteins ; Drug Delivery Systems
Chemical Substances	RNA, Messenger ; Proteins
Language	English
Publishing date	2023-12-12
Publishing country	Germany
Document type	Journal Article ; Review
ZDB-ID	1474949-X
ISSN	1521-4095 ; 0935-9648
ISSN (online)	1521-4095
ISSN	0935-9648
DOI	10.1002/adma.202307822
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Article ; Online: A Comprehensive Review on Intracellular Delivery.

Morshedi Rad, Dorsa / Alsadat Rad, Maryam / Razavi Bazaz, Sajad / Kashaninejad, Navid / Jin, Dayong / Ebrahimi Warkiani, Majid

Advanced materials (Deerfield Beach, Fla.)

2021 Volume 33, Issue 13, Page(s) e2005363

Abstract: ... inside the cells. Furthermore, the applications and challenges of the established and emerging delivery approaches ... differentiates macro-, micro-, and nanoengineered approaches for intracellular delivery. The macroengineered ... from medical applications (cell-based therapy) to fundamental (genome-editing) and industrial (biomanufacture ...

Abstract	Intracellular delivery is considered an indispensable process for various studies, ranging from medical applications (cell-based therapy) to fundamental (genome-editing) and industrial (biomanufacture) approaches. Conventional macroscale delivery systems critically suffer from such issues as low cell viability, cytotoxicity, and inconsistent material delivery, which have opened up an interest in the development of more efficient intracellular delivery systems. In line with the advances in microfluidics and nanotechnology, intracellular delivery based on micro- and nanoengineered platforms has progressed rapidly and held great promises owing to their unique features. These approaches have been advanced to introduce a smorgasbord of diverse cargoes into various cell types with the maximum efficiency and the highest precision. This review differentiates macro-, micro-, and nanoengineered approaches for intracellular delivery. The macroengineered delivery platforms are first summarized and then each method is categorized based on whether it employs a carrier- or membrane-disruption-mediated mechanism to load cargoes inside the cells. Second, particular emphasis is placed on the micro- and nanoengineered advances in the delivery of biomolecules inside the cells. Furthermore, the applications and challenges of the established and emerging delivery approaches are summarized. The topic is concluded by evaluating the future perspective of intracellular delivery toward the micro- and nanoengineered approaches.
MeSH term(s)	Animals ; Drug Delivery Systems/methods ; Humans ; Intracellular Space/metabolism ; Nanotechnology
Language	English
Publishing date	2021-02-16
Publishing country	Germany
Document type	Journal Article ; Review
ZDB-ID	1474949-X
ISSN	1521-4095 ; 0935-9648
ISSN (online)	1521-4095
ISSN	0935-9648
DOI	10.1002/adma.202005363
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Article ; Online: Intracellular Delivery of Functional Proteins with DNA-Protein Nanogels-Lipids Complex.

Mariconti, Marina / Dechamboux, Laurie / Heckmann, Marion / Gros, Julien / Morel, Mathieu / Escriou, Virginie / Baigl, Damien / Hoffmann, Céline / Rudiuk, Sergii

Journal of the American Chemical Society

2024 Volume 146, Issue 8, Page(s) 5118–5127

Abstract: Using functional proteins for therapeutic purposes due to their high selectivity and/or catalytic ... inside living cells remains a major challenge. In contrast, intracellular delivery of nucleic acids has become ... complexation with cationic lipid vectors. We use this approach for delivery of alkaline phosphatase enzyme ...

Abstract	Using functional proteins for therapeutic purposes due to their high selectivity and/or catalytic properties can enable the control of various cellular processes; however, the transport of active proteins inside living cells remains a major challenge. In contrast, intracellular delivery of nucleic acids has become a routine method for a number of applications in gene therapy, genome editing, or immunization. Here we report a functionalizable platform constituting of DNA-protein nanogel carriers cross-linked through streptavidin-biotin or streptactin-biotin interactions and demonstrate its applicability for intracellular delivery of active proteins. We show that the nanogels can be loaded with proteins bearing either biotin, streptavidin, or strep-tag, and the resulting functionalized nanogels can be delivered into living cells after complexation with cationic lipid vectors. We use this approach for delivery of alkaline phosphatase enzyme, which is shown to keep its catalytic activity after internalization by mouse melanoma B16 cells, as demonstrated by the DDAO-phosphate assay. The resulting functionalized nanogels have dimensions on the order of 100 nm, contain around 100 enzyme molecules, and are shown to be transfectable at low lipid concentrations (charge ratio
MeSH term(s)	Animals ; Mice ; Nanogels ; Biotin ; Streptavidin ; Polyethylene Glycols ; Proteins ; DNA/metabolism ; Lipids ; Drug Carriers
Chemical Substances	Nanogels ; Biotin (6SO6U10H04) ; Streptavidin (9013-20-1) ; Polyethylene Glycols (3WJQ0SDW1A) ; Proteins ; DNA (9007-49-2) ; Lipids ; Drug Carriers
Language	English
Publishing date	2024-02-16
Publishing country	United States
Document type	Journal Article
ZDB-ID	3155-0
ISSN	1520-5126 ; 0002-7863
ISSN (online)	1520-5126
ISSN	0002-7863
DOI	10.1021/jacs.3c08000
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Article ; Online: Biomaterials-mediated CRISPR/Cas9 delivery

Ankit Kumar Dubey / Ebrahim Mostafavi

Frontiers in Chemistry, Vol

recent challenges and opportunities in gene therapy

2023 Volume 11

Abstract: ... target effects. However, challenges remain, including optimizing delivery efficiency, reducing off-target ... revolutionizes gene therapy and infectious disease treatment, offering precise and safe editing capabilities ... illnesses by precisely modifying pathogen genomes, and reducing their pathogenicity. Biomaterials facilitate ...

Abstract	The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases holds tremendous potential. This innovative approach combines the advantages of CRISPR/Cas9 with the protective properties of biomaterials, enabling accurate and efficient gene editing while enhancing safety. Biomaterials play a vital role in shielding CRISPR/Cas9 components, such as lipid nanoparticles or viral vectors, from immunological processes and degradation, extending their effectiveness. By utilizing the flexibility of biomaterials, tailored systems can be designed to address specific genetic diseases, paving the way for personalized therapeutics. Furthermore, this delivery method offers promising avenues in combating viral illnesses by precisely modifying pathogen genomes, and reducing their pathogenicity. Biomaterials facilitate site-specific gene modifications, ensuring effective delivery to infected cells while minimizing off-target effects. However, challenges remain, including optimizing delivery efficiency, reducing off-target effects, ensuring long-term safety, and establishing scalable production techniques. Thorough research, pre-clinical investigations, and rigorous safety evaluations are imperative for successful translation from the laboratory to clinical applications. In this review, we discussed how CRISPR/Cas9 delivery using biomaterials revolutionizes gene therapy and infectious disease treatment, offering precise and safe editing capabilities with the potential to significantly improve human health and quality of life.
Keywords	biomaterials ; CRiSPR/Cas ; targeted delivery ; gene therapy ; synthesis ; Chemistry ; QD1-999
Subject code	610
Language	English
Publishing date	2023-09-01T00:00:00Z
Publisher	Frontiers Media S.A.
Document type	Article ; Online
Database	BASE - Bielefeld Academic Search Engine (life sciences selection)

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Article ; Online: Polymeric Delivery of Therapeutic Nucleic Acids.

Kumar, Ramya / Santa Chalarca, Cristiam F / Bockman, Matthew R / Bruggen, Craig Van / Grimme, Christian J / Dalal, Rishad J / Hanson, Mckenna G / Hexum, Joseph K / Reineke, Theresa M

Chemical reviews

2021 Volume 121, Issue 18, Page(s) 11527–11652

Abstract: The advent of genome editing has transformed the therapeutic landscape for several debilitating ... Chemically defined polymers can remediate technological, regulatory, and clinical challenges associated ... that interdisciplinary approaches and methodologies motivated by fundamental questions are key to designing high ...

Abstract	The advent of genome editing has transformed the therapeutic landscape for several debilitating diseases, and the clinical outlook for gene therapeutics has never been more promising. The therapeutic potential of nucleic acids has been limited by a reliance on engineered viral vectors for delivery. Chemically defined polymers can remediate technological, regulatory, and clinical challenges associated with viral modes of gene delivery. Because of their scalability, versatility, and exquisite tunability, polymers are ideal biomaterial platforms for delivering nucleic acid payloads efficiently while minimizing immune response and cellular toxicity. While polymeric gene delivery has progressed significantly in the past four decades, clinical translation of polymeric vehicles faces several formidable challenges. The aim of our Account is to illustrate diverse concepts in designing polymeric vectors towards meeting therapeutic goals of in vivo and ex vivo gene therapy. Here, we highlight several classes of polymers employed in gene delivery and summarize the recent work on understanding the contributions of chemical and architectural design parameters. We touch upon characterization methods used to visualize and understand events transpiring at the interfaces between polymer, nucleic acids, and the physiological environment. We conclude that interdisciplinary approaches and methodologies motivated by fundamental questions are key to designing high-performing polymeric vehicles for gene therapy.
MeSH term(s)	Gene Editing ; Gene Transfer Techniques ; Genetic Therapy/methods ; Nucleic Acids ; Polymers/chemistry
Chemical Substances	Nucleic Acids ; Polymers
Language	English
Publishing date	2021-05-03
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Research Support, U.S. Gov't, Non-P.H.S.
ZDB-ID	207949-5
ISSN	1520-6890 ; 0009-2665
ISSN (online)	1520-6890
ISSN	0009-2665
DOI	10.1021/acs.chemrev.0c00997
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Article ; Online: Delivery challenges for CRISPR-Cas9 genome editing for Duchenne muscular dystrophy.

Padmaswari, Made Harumi / Agrawal, Shilpi / Jia, Mary S / Ivy, Allie / Maxenberger, Daniel A / Burcham, Landon A / Nelson, Christopher E

Biophysics reviews

2023 Volume 4, Issue 1, Page(s) 11307

Abstract: ... for DMD including key summaries of current approaches, delivery methodologies, and the challenges ... CRISPR for gene therapy in humans still abound, including safety and efficiency of delivery, the future ... four exon-skipping drugs receiving conditional Food and Drug Administration approval. However, to date ...

Abstract	Duchene muscular dystrophy (DMD) is an X-linked neuromuscular disorder that affects about one in every 5000 live male births. DMD is caused by mutations in the gene that codes for dystrophin, which is required for muscle membrane stabilization. The loss of functional dystrophin causes muscle degradation that leads to weakness, loss of ambulation, cardiac and respiratory complications, and eventually, premature death. Therapies to treat DMD have advanced in the past decade, with treatments in clinical trials and four exon-skipping drugs receiving conditional Food and Drug Administration approval. However, to date, no treatment has provided long-term correction. Gene editing has emerged as a promising approach to treating DMD. There is a wide range of tools, including meganucleases, zinc finger nucleases, transcription activator-like effector nucleases, and, most notably, RNA-guided enzymes from the bacterial adaptive immune system clustered regularly interspaced short palindromic repeats (CRISPR). Although challenges in using CRISPR for gene therapy in humans still abound, including safety and efficiency of delivery, the future for CRISPR gene editing for DMD is promising. This review will summarize the progress in CRISPR gene editing for DMD including key summaries of current approaches, delivery methodologies, and the challenges that gene editing still faces as well as prospective solutions.
Language	English
Publishing date	2023-02-21
Publishing country	United States
Document type	Journal Article ; Review
ISSN	2688-4089
ISSN (online)	2688-4089
DOI	10.1063/5.0131452
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Article ; Online: CRISPR/Cas9 systems: Delivery technologies and biomedical applications.

Du, Yimin / Liu, Yanfei / Hu, Jiaxin / Peng, Xingxing / Liu, Zhenbao

Asian journal of pharmaceutical sciences

2023 Volume 18, Issue 6, Page(s) 100854

Abstract: ... the key factors affecting the delivery process and the current challenges facing the CRISPR/Cas9 system ... vector, and non-viral vector delivery strategies, including plasmid-, mRNA- and protein-based approach ... of this potent gene-editing tool, ensuring efficient and secure delivery to the target site is paramount ...

Abstract	The emergence of the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) genome-editing system has brought about a significant revolution in the realm of managing human diseases, establishing animal models, and so on. To fully harness the potential of this potent gene-editing tool, ensuring efficient and secure delivery to the target site is paramount. Consequently, developing effective delivery methods for the CRISPR/Cas9 system has become a critical area of research. In this review, we present a comprehensive outline of delivery strategies and discuss their biomedical applications in the CRISPR/Cas9 system. We also provide an in-depth analysis of physical, viral vector, and non-viral vector delivery strategies, including plasmid-, mRNA- and protein-based approach. In addition, we illustrate the biomedical applications of the CRISPR/Cas9 system. This review highlights the key factors affecting the delivery process and the current challenges facing the CRISPR/Cas9 system, while also delineating future directions and prospects that could inspire innovative delivery strategies. This review aims to provide new insights and ideas for advancing CRISPR/Cas9-based delivery strategies and to facilitate breakthroughs in biomedical research and therapeutic applications.
Language	English
Publishing date	2023-10-21
Publishing country	Netherlands
Document type	Journal Article ; Review
ZDB-ID	2650931-3
ISSN	2221-285X ; 1818-0876 ; 2221-285X
ISSN (online)	2221-285X
ISSN	1818-0876 ; 2221-285X
DOI	10.1016/j.ajps.2023.100854
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