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Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease

Shuling Guo Antisense Drug Discovery; Isis Pharmaceuticals; Carlsbad, CA USA

Sheri L Booten Antisense Drug Discovery; Isis Pharmaceuticals; Carlsbad, CA USA

Andrew Watt Antisense Drug Discovery; Isis Pharmaceuticals; Carlsbad, CA USA

Luis Alvarado Antisense Drug Discovery; Isis Pharmaceuticals; Carlsbad, CA USA

Susan M Freier Antisense Drug Discovery; Isis Pharmaceuticals; Carlsbad, CA USA

Jeffery H Teckman Department of Pediatrics; St. Louis University School of Medicine; St. Louis, MO USA

Michael L McCaleb Antisense Drug Discovery; Isis Pharmaceuticals; Carlsbad, CA USA

Brett P Monia Antisense Drug Discovery; Isis Pharmaceuticals; Carlsbad, CA USACorrespondence[email protected]

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Article: e28511 | Received 27 Jan 2014, Accepted 11 Mar 2014, Published online: 12 Mar 2014

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https://doi.org/10.4161/rdis.28511
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Nélia Abreu, Vítor Magno Pereira, Madalena Pestana & Luís Jasmins. (2023) Future Perspectives in the Diagnosis and Treatment of Liver Disease Associated with Alpha-1 Antitrypsin Deficiency. GE - Portuguese Journal of Gastroenterology, pages 1-9.
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Filipa Bezerra, Christoph Niemietz, Hartmut H. J. Schmidt, Andree Zibert, Shuling Guo, Brett P. Monia, Paula Gonçalves, Maria João Saraiva & Maria Rosário Almeida. (2021) In Vitro and In Vivo Effects of SerpinA1 on the Modulation of Transthyretin Proteolysis. International Journal of Molecular Sciences 22:17, pages 9488.
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Katharina Remih, Samira Amzou & Pavel Strnad. (2021) Alpha1-antitrypsin deficiency: New therapies on the horizon. Current Opinion in Pharmacology 59, pages 149-156.
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Kyle Stearns, Monica Goldklang, Rui Xiao, Tina Zelonina, Keith Blomenkamp, Jeffery Teckman & Jeanine M. D’Armiento. (2021) Knockdown of Alpha-1 Antitrypsin with antisense oligonucleotide does not exacerbate smoke induced lung injury. PLOS ONE 16:2, pages e0246040.
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Rashika Joshi, Mohit Ojha, Jana Lewis, Qiang Fan, Brett Monia, Shuling Guo & Brian M. Varisco. (2019) Sex-specific differences in emphysema using a murine antisense oligonucleotide model of α-1 antitrypsin deficiency. American Journal of Physiology-Lung Cellular and Molecular Physiology 316:6, pages L1165-L1173.
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Rashika Joshi, Andrea Heinz, Qiang Fan, Shuling Guo, Brett Monia, Christian E. H. Schmelzer, Anthony S. Weiss, Matthew Batie, Harikrishnan Parameshwaran & Brian M. Varisco. (2018) Role for Cela1 in Postnatal Lung Remodeling and Alpha-1 Antitrypsin–Deficient Emphysema . American Journal of Respiratory Cell and Molecular Biology 59:2, pages 167-178.
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Dolan SondhiKatie M. StilesBishnu P. DeRonald G. Crystal. (2017) Genetic Modification of the Lung Directed Toward Treatment of Human Disease. Human Gene Therapy 28:1, pages 3-84.
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Ignacio Blanco. 2017. Blanco's Overview of Alpha-1 Antitrypsin Deficiency. Blanco's Overview of Alpha-1 Antitrypsin Deficiency 207 225 .

Mariam Aghajan, Shuling Guo & Brett P. Monia. 2017. Alpha-1 Antitrypsin Deficiency. Alpha-1 Antitrypsin Deficiency 127 138 .

Maria J. Chiuchiolo & Ronald G. Crystal. (2016) Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease. Annals of the American Thoracic Society 13:Supplement_4, pages S352-S369.
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K.I. Papadopoulos, P. Wattanaarsakit, W. Prasongchean & R. Narain. 2016. Polymers and Nanomaterials for Gene Therapy. Polymers and Nanomaterials for Gene Therapy 231 256 .

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Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease

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Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease

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