Article
Biotechnology & Applied Microbiology
Anna Voznesenskaya, Per-Olof Berggren, Erwin Ilegems
Summary: This study characterized the transduction performance of AAV serotype 8 (AAV8) and human adenovirus type 5 (AdV5) during pseudoislet formation. The results provide a reference for future studies using AAVs for gene transfer to islet cell organoids and facilitate the development of high-quality engineered pseudoislets for clinical transplantation.
FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY
(2023)
Article
Biochemistry & Molecular Biology
Meera Vyas, Natalie M. Deschenes, Karlaina J. L. Osmon, Zhilin Chen, Imtiaz Ahmad, Shalini Kot, Patrick Thompson, Chris Richmond, Steven J. Gray, Jagdeep S. Walia
Summary: This study assessed the effects of gene therapy in an ABGM2 mouse model and found that it led to a reduction in GM2 accumulation and long-term persistence of vector genomes in the brain and liver.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Biotechnology & Applied Microbiology
Benjamin Stolte, Olivia Schreiber-Katz, Rene Guenther, Claudia Diana Wurster, Susanne Petri, Alma Osmanovic, Maren Freigang, Zeljko Uzelac, Markus Leo, Otgonzul von Velsen, Wibke Bayer, Ulf Dittmer, Christoph Kleinschnitz, Tim Hagenacker
Summary: This study investigated the prevalence of anti-AAV9 antibodies in adult patients with SMA and found that only a small percentage of patients had elevated antibody titers. This suggests that gene therapy with AAV9 vectors may be feasible in adult SMA patients.
HUMAN GENE THERAPY
(2022)
Article
Biotechnology & Applied Microbiology
R. Ashton Lavoie, Jeffrey T. Zugates, Andrew T. Cheeseman, Matt A. Teten, Srivatsan Ramesh, Julia M. Freeman, Summer Swango, Jeremy Fitzpatrick, Amod Joshi, Bradley Hollers, Zufan Debebe, Tyler K. Lindgren, Amber N. Kozak, Vinay K. Kondeti, Mary K. Bright, Eric J. Yearley, Alexander Tracy, Jacob A. Irwin, Michael Guerrero
Summary: Adeno-associated virus-based gene therapies have shown great potential in treating genetic disorders. However, achieving high resolution between full and empty capsids is challenging. Anion exchange chromatography has emerged as a popular method for full capsid enrichment, but further improvements are needed.
BIOTECHNOLOGY AND BIOENGINEERING
(2023)
Article
Medicine, Research & Experimental
Kelsey R. Pflepsen, Cristina D. Peterson, Kelley F. Kitto, Maureen S. Riedl, R. Scott McIvor, George L. Wilcox, Lucy Vulchanova, Carolyn A. Fairbanks
Summary: The distribution and kinetics of AAV5 vector following lumbar intrathecal injection in mice were evaluated in this study. Results showed rapid distribution of AAV5 particles to all levels of the spinal cord and dorsal root ganglia, with preferential targeting of DRG tissue and possible clearance mechanisms via DRG macrophages.
MOLECULAR PHARMACEUTICS
(2021)
Article
Gastroenterology & Hepatology
Nadja Meumann, Marti Cabanes-Creus, Moritz Ertelt, Renina Gale Navarro, Julie Lucifora, Qinggong Yuan, Karin Nien-Huber, Ahmed Abdelrahman, Xuan-Khang Vu, Liang Zhang, Ann-Christin Franke, Christian Schmithals, Albrecht Piiper, Annabelle Vogt, Maria Gonzalez-Carmona, Jochen T. Frueh, Evelyn Ullrich, Philip Meuleman, Steven R. Talbot, Margarete Odenthal, Michael Ott, Erhard Seifried, Clara T. Schoeder, Joachim Schwable, Leszek Lisowski, Hildegard Buning
Summary: This article reports two gene vector variants, MLIV.K and MLIV.A, which were obtained through in vivo AAV peptide display selection in mice. They showed improved hepatocyte targeting and transduction efficiency compared to AAV2 and AAV8, and have potential for liver disease treatment.
Article
Cell Biology
Jinliang Li, Shannon C. Kelly, Jan R. Ivey, Pamela K. Thorne, Kelly P. Yamada, Tadao Aikawa, Renata Mazurek, James R. Turk, Kleiton Augusto Santos Silva, Amira R. Amin, Darla L. Tharp, Christina M. Mueller, Hrishikesh Thakur, Emily Leary, Timothy L. Domeier, R. Scott Rector, Kenneth Fish, Federico Cividini, Kiyotake Ishikawa, Craig A. Emter, Michael S. Kapiloff
Summary: This study assessed the biodistribution of self-complementary serotype 9 adeno-associated virus (AAV9sc) biologics in swine, demonstrating successful targeted delivery of cardiomyocyte-selective viral vectors without toxic effects. The results indicate the potential efficacy of these vectors in preclinical experimental settings.
PHYSIOLOGICAL GENOMICS
(2022)
Article
Medicine, Research & Experimental
Agostina Carestia, Seok-Joo Kim, Franziska Horling, Hanspeter Rottensteiner, Christian Lubich, Birgit M. Reipert, Brian A. Crowe, Craig N. Jenne
Summary: In this study, the immune response mediated by an AAV vector in a mouse model was demonstrated. AAV8 infection resulted in a moderate but significant activation of the immune system in the liver, involving platelet aggregation, NET formation, and the recruitment of immune cells. Kupffer cells were essential to initiate this immune response, while the death of liver cells caused by AAV8 did not lead to a robust, sustained inflammatory response. These findings suggest AAV8 is a suitable vector for gene therapy.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2021)
Article
Endocrinology & Metabolism
Yuka Kinoshita, Fatma F. Mohamed, Flavia Amadeu de Oliveira, Sonoko Narisawa, Koichi Miyake, Brian L. Foster, Jose Luis Millan
Summary: Hypophosphatasia (HPP) is a disease caused by loss-of-function mutations in the ALPL gene encoding tissue-nonspecific alkaline phosphatase (TNAP). Currently, enzyme replacement therapy (ERT) and gene therapy are the main methods for treating the disease. Gene therapy may be a promising alternative treatment that can significantly extend patient lifespan and improve their skeletal and dental conditions.
JOURNAL OF BONE AND MINERAL RESEARCH
(2021)
Article
Immunology
Chenjuan Shi, Li Tian, Wenwen Zheng, Yelei Zhu, Peilu Sun, Lele Liu, Wenkai Liu, Yanyan Song, Xianzhu Xia, Xianghong Xue, Xuexing Zheng
Summary: The study found that AAV9-RABVG is a single-dose vaccine candidate that can induce robust, long-term humoral responses and Th1 and Th2 cell-mediated immune responses in mice and non-human primates.
EMERGING MICROBES & INFECTIONS
(2022)
Article
Multidisciplinary Sciences
Tobias P. Woerner, Antonette Bennett, Sana Habka, Joost Snijder, Olga Friese, Thomas Powers, Mavis Agbandje-McKenna, Albert J. R. Heck
Summary: Recent research on Adeno-associated viruses (AAVs) using high-resolution native mass spectrometry has revealed that the capsids of different AAV serotypes assemble stochastically, leading to a highly heterogeneous population of capsids with varying compositions. Even the most abundant VP stoichiometry represents only a small percentage of the total AAV population. The systematic scoring of simulations against experimental native MS data offers a sensitive new method to characterize these therapeutically important heterogeneous capsids.
NATURE COMMUNICATIONS
(2021)
Article
Microbiology
Edward E. Large, Michael S. Chapman
Summary: Adeno-associated viruses (AAV) are widely used for gene therapy, and monoclonal antibodies against various AAV serotypes have been developed. Previous studies suggested that neutralizing antibodies inhibit binding to glycan receptors or interfere with post-entry steps. However, recent research has identified a protein receptor and revealed that antibody interference with protein receptor binding may be the predominant mechanism of neutralization.
FRONTIERS IN MICROBIOLOGY
(2023)
Article
Biochemistry & Molecular Biology
Jinxin Fan, Eduardo Barbieri, Shriarjun Shastry, Stefano Menegatti, Cristiana Boi, Ruben G. Carbonell
Summary: This study developed nonwoven ion-exchange membrane adsorbents for efficient purification of AAV2 from cell lysate and achieved high purity by utilizing a series of two different ion-exchange membranes.
Review
Clinical Neurology
Katarzyna Kotulska, Aviva Fattal-Valevski, Jana Haberlova
Summary: Spinal muscular atrophy is a neuromuscular disease caused by mutation of the SMN1 gene, but disease modifying therapies have shown effectiveness in improving motor functions of patients with SMA.
FRONTIERS IN NEUROLOGY
(2021)
Article
Genetics & Heredity
Wuh-Liang Hwu, Shin-Ichi Muramatsu, Bruria Gidoni-Ben-Zeev
Summary: This article illustrates the low risk of immune response to AAV serotype 2 vector-mediated gene therapy to the brain with support from clinical trial data in aromatic L-amino acid decarboxylase deficiency and Parkinson disease.
CURRENT GENE THERAPY
(2022)
Article
Cardiac & Cardiovascular Systems
Francesco Villa, Albino Carrizzo, Chiara C. Spinelli, Anna Ferrario, Alberto Malovini, Anna Maciag, Antonio Damato, Alberto Auricchio, Gaia Spinetti, Elena Sangalli, Zexu Dang, Michele Madonna, Mariateresa Ambrosio, Leopoldo Sitia, Paolo Bigini, Gaetano Cali, Stefan Schreiber, Thomas Perls, Sergio Fucile, Francesca Mulas, Almut Nebel, Riccardo Bellazzi, Paolo Madeddu, Carmine Vecchione, Annibale A. Puca
CIRCULATION RESEARCH
(2015)
Article
Biotechnology & Applied Microbiology
Rita Ferla, Pamela Claudiani, Marco Savarese, Karen Kozarsky, Rossella Parini, Maurizio Scarpa, Maria Alice Donati, Giovanni Sorge, John J. Hopwood, Giancarlo Parenti, Simona Fecarotta, Vincenzo Nigro, Hatice Serap Sivri, Ans Van der Ploeg, Generoso Andria, Nicola Brunetti-Pierri, Alberto Auricchio
HUMAN GENE THERAPY
(2015)
Review
Biotechnology & Applied Microbiology
Ivana Trapani, Sandro Banfi, Francesca Simonelli, Enrico M. Surace, Alberto Auricchio
HUMAN GENE THERAPY
(2015)
Article
Cell Biology
Marzia Di Donato, Antonio Bilancio, Loredana D'Amato, Pamela Claudiani, Maria Antonietta Oliviero, Maria Vittoria Barone, Alberto Auricchio, Ettore Appella, Antimo Migliaccio, Ferdinando Auricchio, Gabriella Castoria
MOLECULAR BIOLOGY OF THE CELL
(2015)
Article
Biotechnology & Applied Microbiology
Maria Grazia Biferi, Carmine Nicoletti, Germana Falcone, Eleonora M. R. Puggioni, Nunzia Passaro, Alessia Mazzola, Deborah Pajalunga, Germana Zaccagnini, Emanuele Rizzuto, Alberto Auricchio, Lorena Zentilin, Gabriele De Luca, Mauro Giacca, Fabio Martelli, Antonio Musio, Antonio Musaro, Marco Crescenzi
Article
Medicine, Research & Experimental
Alberto Auricchio, Ivana Trapani, Rando Allikmets
COLD SPRING HARBOR PERSPECTIVES IN MEDICINE
(2015)
Article
Biochemistry & Molecular Biology
A. Puppo, G. Cesi, E. Marrocco, P. Piccolo, S. Jacca, D. M. Shayakhmetov, R. J. Parks, B. L. Davidson, S. Colloca, N. Brunetti-Pierri, P. Ng, G. Donofrio, A. Auricchio
Article
Biochemistry & Molecular Biology
P. Colella, I. Trapani, G. Cesi, A. Sommella, A. Manfredi, A. Puppo, C. Iodice, S. Rossi, F. Simonelli, M. Giunti, M. L. Bacci, A. Auricchio
Article
Biotechnology & Applied Microbiology
Rita Ferla, Pamela Claudiani, Gabriella Cotugno, Paola Saccone, Elvira De Leonibus, Alberto Auricchio
HUMAN GENE THERAPY
(2014)
Article
Biotechnology & Applied Microbiology
Eduard Ayuso, Veronique Blouin, Martin Lock, Susan McGorray, Xavier Leon, Mauricio R. Alvira, Alberto Auricchio, Stephanie Bucher, Abdelwahed Chtarto, K. Reed Clark, Christophe Darmon, Monica Doria, Will Fountain, Guangping Gao, Kai Gao, Mauro Giacca, Juergen Kleinschmidt, Barbara Leuchs, Catherine Melas, Hiroaki Mizukami, Marcus Mueller, Yvet Noordman, Olivier Bockstael, Keiya Ozawa, Catherine Pythoud, Marina Sumaroka, Richard Surosky, Liliane Tenenbaum, Inge van der Linden, Brigitte Weins, J. Fraser Wright, Xinhua Zhang, Lorena Zentilin, Fatima Bosch, Richard O. Snyder, Philippe Moullier
HUMAN GENE THERAPY
(2014)
Article
Biochemistry & Molecular Biology
Marianna Marino, Tatiana Stoilova, Carlotta Giorgi, Angela Bachi, Angela Cattaneo, Alberto Auricchio, Paolo Pinton, Ester Zito
HUMAN MOLECULAR GENETICS
(2015)
Correction
Medicine, Research & Experimental
Marinella Pirozzi, Angelo Quattrini, Gennaro Andolfi, Giorgia Dina, Maria Chiara Malaguti, Alberto Auricchio, Elena I. Rugarli
JOURNAL OF CLINICAL INVESTIGATION
(2014)
Article
Multidisciplinary Sciences
Paola Saccone, Gabriella Cotugno, Fabio Russo, Rosa Mastrogiacomo, Alessandra Tessitore, Alberto Auricchio, Elvira De Leonibus
SCIENTIFIC REPORTS
(2014)
Editorial Material
Biochemistry & Molecular Biology
Ivana Trapani, Patrizia Tornabene, Alberto Auricchio
Article
Multidisciplinary Sciences
Patrizia Tornabene, Rita Ferla, Manel Llado-Santaeularia, Miriam Centrulo, Margherita Dell'Anno, Federica Esposito, Elena Marrocco, Emanuela Pone, Renato Minopoli, Carolina Iodice, Edoardo Nusco, Settimio Rossi, Hristiana Lyubenova, Anna Manfredi, Lucio Di Filippo, Antonella Iuliano, Annalaura Torella, Giulio Piluso, Francesco Musacchia, Enrico Maria Surace, Davide Cacchiarelli, Vincenzo Nigro, Alberto Auricchio
Summary: The study introduces a novel gene therapy approach, AAV-HITI, to overcome challenges in gene therapy. The results show that AAV-HITI enables efficient gene integration in both retina and liver, leading to therapeutic benefits for genetic diseases.
NATURE COMMUNICATIONS
(2022)
