Reducing TMPRSS6 ameliorates hemochromatosis and β-thalassemia in mice

door Guo, Shuling, Casu, Carla, Gardenghi, Sara, Booten, Sheri, Aghajan, Mariam, Peralta, Raechel, Watt, Andy, Freier, Sue, Monia, Brett P., Rivella, Stefano
Gepubliceerd in 2013

Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice

door Tasfaout, Hichem, Buono, Suzie, Guo, Shuling, Kretz, Christine, Messaddeq, Nadia, Booten, Sheri, Greenlee, Sarah, Monia, Brett P., Cowling, Belinda S., Laporte, Jocelyn
Gepubliceerd in Nat Commun (2017)

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

door Guo, Shuling, Booten, Sheri L, Watt, Andrew, Alvarado, Luis, Freier, Susan M, Teckman, Jeffery H, McCaleb, Michael L, Monia, Brett P
Gepubliceerd in 2014

Antisense Reduction of Mutant COMP Reduces Growth Plate Chondrocyte Pathology

door Posey, Karen L., Coustry, Francoise, Veerisetty, Alka C., Hossain, Mohammad, Gattis, Danielle, Booten, Sheri, Alcorn, Joseph L., Seth, Punit P., Hecht, Jacqueline T.
Gepubliceerd in Mol Ther (2017)

Comparison of the pharmacological profiles of murine antisense oligonucleotides targeting apolipoprotein B and microsomal triglyceride transfer protein

door Lee, Richard G., Fu, Wuxia, Graham, Mark J., Mullick, Adam E., Sipe, Donna, Gattis, Danielle, Bell, Thomas A., Booten, Sheri, Crooke, Rosanne M.
Gepubliceerd in 2013

Targeted Delivery of Stk25 Antisense Oligonucleotides to Hepatocytes Protects Mice Against Nonalcoholic Fatty Liver Disease

door Cansby, Emmelie, Nuñez-Durán, Esther, Magnusson, Elin, Amrutkar, Manoj, Booten, Sheri L., Kulkarni, Nagaraj M., Svensson, L. Thomas, Borén, Jan, Marschall, Hanns-Ulrich, Aghajan, Mariam, Mahlapuu, Margit
Gepubliceerd in Cell Mol Gastroenterol Hepatol (2018)

Partial Hepatectomy Induced Long Noncoding RNA Inhibits Hepatocyte Proliferation during Liver Regeneration

door Huang, Lulu, Damle, Sagar S., Booten, Sheri, Singh, Priyam, Sabripour, Mahyar, Hsu, Jeff, Jo, Minji, Katz, Melanie, Watt, Andy, Hart, Christopher E., Freier, Susan M., Monia, Brett P., Guo, Shuling
Gepubliceerd in PLoS One (2015)

Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin–related liver disease in mice

door Guo, Shuling, Booten, Sheri L., Aghajan, Mariam, Hung, Gene, Zhao, Chenguang, Blomenkamp, Keith, Gattis, Danielle, Watt, Andrew, Freier, Susan M., Teckman, Jeffery H., McCaleb, Michael L., Monia, Brett P.
Gepubliceerd in 2013

Serine/threonine protein kinase 25 antisense oligonucleotide treatment reverses glucose intolerance, insulin resistance, and nonalcoholic fatty liver disease in mice

door Nuñez‐Durán, Esther, Aghajan, Mariam, Amrutkar, Manoj, Sütt, Silva, Cansby, Emmelie, Booten, Sheri L., Watt, Andrew, Ståhlman, Marcus, Stefan, Norbert, Häring, Hans‐Ulrich, Staiger, Harald, Borén, Jan, Marschall, Hanns‐Ulrich, Mahlapuu, Margit
Gepubliceerd in Hepatol Commun (2017)

Inhibition of Protein Tyrosine Phosphatase-1B with Antisense Oligonucleotides Improves Insulin Sensitivity and Increases Adiponectin Concentrations in Monkeys

door Swarbrick, Michael M., Havel, Peter J., Levin, Arthur A., Bremer, Andrew A., Stanhope, Kimber L., Butler, Madeline, Booten, Sheri L., Graham, James L., McKay, Robert A., Murray, Susan F., Watts, Lynnetta M., Monia, Brett P., Bhanot, Sanjay
Gepubliceerd in 2009

Antisense oligonucleotide treatment ameliorates IFN-γ–induced proteinuria in APOL1-transgenic mice

door Aghajan, Mariam, Booten, Sheri L., Althage, Magnus, Hart, Christopher E., Ericsson, Anette, Maxvall, Ingela, Ochaba, Joseph, Menschik-Lundin, Angela, Hartleib, Judith, Kuntz, Steven, Gattis, Danielle, Ahlström, Christine, Watt, Andrew T., Engelhardt, Jeffery A., Monia, Brett P., Magnone, Maria Chiara, Guo, Shuling
Gepubliceerd in JCI Insight (2019)

Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data

door Viney, Nicholas J., Guo, Shuling, Tai, Li‐Jung, Baker, Brenda F., Aghajan, Mariam, Jung, Shiangtung W., Yu, Rosie Z., Booten, Sheri, Murray, Heather, Machemer, Todd, Burel, Sebastien, Murray, Sue, Buchele, Gustavo, Tsimikas, Sotirios, Schneider, Eugene, Geary, Richard S., Benson, Merrill D., Monia, Brett P.
Gepubliceerd in ESC Heart Fail (2020)

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice

door Prakash, Thazha P., Graham, Mark J., Yu, Jinghua, Carty, Rick, Low, Audrey, Chappell, Alfred, Schmidt, Karsten, Zhao, Chenguang, Aghajan, Mariam, Murray, Heather F., Riney, Stan, Booten, Sheri L., Murray, Susan F., Gaus, Hans, Crosby, Jeff, Lima, Walt F., Guo, Shuling, Monia, Brett P., Swayze, Eric E., Seth, Punit P.
Gepubliceerd in 2014

Correcting β-thalassemia by combined therapies that restrict iron and modulate erythropoietin activity

door Casu, Carla, Pettinato, Mariateresa, Liu, Alison, Aghajan, Mariam, Lo Presti, Vania, Lidonnici, Maria Rosa, Munoz, Kevin A., O’Hara, Emir, Olivari, Violante, Di Modica, Simona Maria, Booten, Sheri, Guo, Shuling, Neil, Garry, Miari, Reem, Shapir, Nir, Zafir-Lavie, Inbal, Domev, Hagit, Ferrari, Giuliana, Sitara, Despina, Nai, Antonella, Rivella, Stefano
Gepubliceerd in Blood (2020)