Intranasal application of adeno-associated viruses: a systematic review

被引：10

作者：

Gadenstaetter, Anselm J.

Schmutzler, Lukas ^{[1
]}

Grimm, Dirk

Landegger, Lukas D.

机构：

[1] Med Univ Vienna, Vienna Gen Hosp, Dept Otolaryngol, Waehringer Guertel 18-20, A-1090 Vienna, Austria

来源：

TRANSLATIONAL RESEARCH | 2022年 / 248卷

关键词：

HUMAN-PAPILLOMAVIRUS TYPE-16; AIRWAY EPITHELIAL-CELLS; GENE-TRANSFER; IMMUNE-RESPONSES; RECOMBINANT NT4-NAP/AAV; SHEEP RETROVIRUS; NASAL EPITHELIA; CYSTIC-FIBROSIS; OPEN-LABEL; DELIVERY;

D O I：

10.1016/j.trsl.2022.05.002

中图分类号：

R446 [实验室诊断]; R-33 [实验医学、医学实验];

学科分类号：

1001 ;

摘要：

Adeno-associated viruses (AAVs) represent some of the most commonly employed vectors for targeted gene delivery and their extensive study has resulted in the approval of multiple gene therapies to treat human diseases. The intranasal route of vector application in gene therapy offers several advantages over traditional ways of administration. In addition to targeting local tissue like the olfactory epithelium, it provides minimally invasive access to various organ systems, including the central nervous system and the respiratory tract. Through a systematic literature review, a total of 53 articles that investigated the intranasal application of AAVs were identi-fied, included, and summarized in this manuscript. Within these studies, AAV-based gene therapy was mainly investigated for its application in various infectious, pul-monary, or neurologic and/or psychiatric diseases. This review gives a comprehen-sive overview of the current technological state of the art regarding the intranasal application of AAVs for gene transfer and discusses remaining hurdles, which still have to be resolved before this approach can effectively be implemented in the routine clinical setting.

引用

页码：87 / 110

页数：24

共 98 条

[1] Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing [J].

Adachi, Kei ;

Enoki, Tatsuji ;

Kawano, Yasuhiro ;

Veraz, Michael ;

Nakai, Hiroyuki .

NATURE COMMUNICATIONS, 2014, 5

[2] Adeno-Associated Virus 9-Mediated Airway Expression of Antibody Protects Old and Immunodeficient Mice against Influenza Virus [J].

Adam, Virginie S. ;

Crosariol, Marco ;

Kumar, Sachin ;

Ge, Moyar Q. ;

Czack, Sarah E. ;

Roy, Soumitra ;

Haczku, Angela ;

Tretiakova, Anna ;

Wilson, James M. ;

Limberis, Maria P. .

CLINICAL AND VACCINE IMMUNOLOGY, 2014, 21 (11) :1528-1533

[3] Nonintegrating Gene Therapy Vectors [J].

Athanasopoulos, Takis ;

Munye, Mustafa M. ;

Yanez-Munoz, Rafael J. .

HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA, 2017, 31 (05) :753-+

[4] Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins [J].

Auricchio, A ;

O'Connor, E ;

Weiner, D ;

Gao, GP ;

Hildinger, M ;

Wang, LL ;

Calcedo, R ;

Wilson, JM .

JOURNAL OF CLINICAL INVESTIGATION, 2002, 110 (04) :499-504

[5] Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors [J].

Becker, Jonas ;

Stanifer, Megan Lynn ;

Leist, Sarah Rebecca ;

Stolp, Bettina ;

Maiakovska, Olena ;

West, Ande ;

Wiedtke, Ellen ;

Boerner, Kathleen ;

Ghanem, Ali ;

Ambiel, Ina ;

Tse, Longping Victor ;

Fackler, Oliver Till ;

Baric, Ralph Steven ;

Boulant, Steeve ;

Grimm, Dirk .

MOLECULAR THERAPY, 2022, 30 (05) :2005-2023

[6] The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice [J].

Bell, Christie L. ;

Vandenberghe, Luk H. ;

Bell, Peter ;

Limberis, Maria P. ;

Gao, Guang-Ping ;

Van Vliet, Kim ;

Agbandje-McKenna, Mavis ;

Wilson, James M. .

JOURNAL OF CLINICAL INVESTIGATION, 2011, 121 (06) :2427-2435

[7] An optimized protocol for detection of E-coli β-galactosidase in lung tissue following gene transfer [J].

Bell, P ;

Limberis, M ;

Gao, GP ;

Wu, D ;

Bove, MS ;

Sanmiguel, JC ;

Wilson, JM .

HISTOCHEMISTRY AND CELL BIOLOGY, 2005, 124 (01) :77-85

[8] Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I [J].

Belur, Lalitha R. ;

Romero, Megan ;

Lee, Junggu ;

Podetz-Pedersen, Kelly M. ;

Nan, Zhenhong ;

Riedl, Maureen S. ;

Vulchanova, Lucy ;

Kitto, Kelley F. ;

Fairbanks, Carolyn A. ;

Kozarsky, Karen F. ;

Orchard, Paul J. ;

Frey, William H., II ;

Low, Walter C. ;

McIvor, R. Scott .

FRONTIERS IN MOLECULAR NEUROSCIENCE, 2021, 14

[9] Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I [J].

Belur, Lalitha R. ;

Temme, Alexa ;

Podetz-Pedersen, Kelly M. ;

Riedl, Maureen ;

Vulchanova, Lucy ;

Robinson, Nicholas ;

Hanson, Leah R. ;

Kozarsky, Karen F. ;

Orchard, Paul J. ;

Frey, William H., II ;

Low, Walter C. ;

McIvor, R. Scott .

HUMAN GENE THERAPY, 2017, 28 (07) :576-587

[10] Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round Screening [J].

Boerner, Kathleen ;

Kienle, Eike ;

Huang, Lin-Ya ;

Weinmann, Jonas ;

Sacher, Anna ;

Bayer, Philipp ;

Stullein, Christian ;

Fakhiri, Julia ;

Zimmermann, Laura ;

Westhaus, Adrian ;

Beneke, Juergen ;

Beil, Nina ;

Wiedtke, Ellen ;

Schmelas, Carolin ;

Miltner, Dominik ;

Rau, Alexander ;

Erfle, Holger ;

Kraeusslich, Hans-Georg ;

Mueller, Martin ;

Agbandje-McKenna, Mavis ;

Grimm, Dirk .

MOLECULAR THERAPY, 2020, 28 (04) :1016-1032

← 1 2 3 4 5 6 7 8 9 10 →