Clinical applications of gene therapy for rare diseases: A review

por Papaioannou, Ioannis, Owen, James S., Yáñez‐Muñoz, Rafael J.
Publicado 2023

Corticospinal tract transduction: a comparison of seven adeno-associated viral vector serotypes and a non-integrating lentiviral vector: Transduction of the CST using viral vectors

por Hutson, Thomas H., Verhaagen, Joost, Yáñez-Muñoz, Rafael J., Moon, Lawrence D.F.
Publicado 2011

Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

por Chilcott, Ellie M., Muiruri, Evalyne W., Hirst, Theodore C., Yáñez-Muñoz, Rafael J.
Publicado 2021

Correction: Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

por Chilcott, Ellie M., Muiruri, Evalyne W., Hirst, Theodore C., Yáñez-Muñoz, Rafael J.
Publicado 2022

Genomic insertion of lentiviral DNA circles directed by the yeast Flp recombinase

por Moldt, Brian, Staunstrup, Nicklas H, Jakobsen, Maria, Yáñez-Muñoz, Rafael J, Mikkelsen, Jacob G
Publicado 2008

High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors

por Ahmed, Sherif G., Waddington, Simon N., Boza-Morán, Maria Gabriela, Yáñez-Muñoz, Rafael J.
Publicado 2018

Cortical Overexpression of Neuronal Calcium Sensor-1 Induces Functional Plasticity in Spinal Cord Following Unilateral Pyramidal Tract Injury in Rat

por Yip, Ping K., Wong, Liang-Fong, Sears, Thomas A., Yáñez-Muñoz, Rafael J., McMahon, Stephen B.
Publicado 2010

An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

por Selvakumaran, Jamuna, Ursu, Simona, Bowerman, Melissa, Lu-Nguyen, Ngoc, Wood, Matthew J., Malerba, Alberto, Yáñez-Muñoz, Rafael J.
Publicado 2023

Therapeutic strategies for spinal muscular atrophy: SMN and beyond

por Bowerman, Melissa, Becker, Catherina G., Yáñez-Muñoz, Rafael J., Ning, Ke, Wood, Matthew J. A., Gillingwater, Thomas H., Talbot, Kevin
Publicado 2017

Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

por Ahlskog, Nina, Hayler, Daniel, Krueger, Anja, Kubinski, Sabrina, Claus, Peter, Hammond, Suzan M., Wood, Matthew J. A., Yáñez-Muñoz, Rafael J., Bowerman, Melissa
Publicado 2020

AAV9-mediated SMN gene therapy rescues cardiac desmin but not lamin A/C and elastin dysregulation in Smn(2B/−) spinal muscular atrophy mice

por Brown, Sharon J, Šoltić, Darija, Synowsky, Silvia A, Shirran, Sally L, Chilcott, Ellie, Shorrock, Hannah K, Gillingwater, Thomas H, Yáñez-Muñoz, Rafael J, Schneider, Bernard, Bowerman, Melissa, Fuller, Heidi R
Publicado 2023

Decay in survival motor neuron and plastin 3 levels during differentiation of iPSC-derived human motor neurons

por Boza-Morán, María G, Martínez-Hernández, Rebeca, Bernal, Sara, Wanisch, Klaus, Also-Rallo, Eva, Le Heron, Anita, Alías, Laura, Denis, Cécile, Girard, Mathilde, Yee, Jiing-Kuan, Tizzano, Eduardo F., Yáñez-Muñoz, Rafael J
Publicado 2015

Induction of the cell survival kinase Sgk1: A possible novel mechanism for α-phenyl-N-tert-butyl nitrone in experimental stroke

por McCaig, Catherine, Ataliotis, Paris, Shtaya, Anan, Omar, Ayan S, Green, A Richard, Kind, Clive N, Pereira, Anthony C, Naray-Fejes-Toth, Aniko, Fejes-Toth, Geza, Yáñez-Muñoz, Rafael J, Murray, James T, Hainsworth, Atticus H
Publicado 2017

Lentiviral vectors express chondroitinase ABC in cortical projections and promote sprouting of injured corticospinal axons

por Zhao, Rong-Rong, Muir, Elizabeth M., Alves, João Nuno, Rickman, Hannah, Allan, Anna Y., Kwok, Jessica C., Roet, Kasper C.D., Verhaagen, Joost, Schneider, Bernard L., Bensadoun, Jean-Charles, Ahmed, Sherif G., Yáñez-Muñoz, Rafael J., Keynes, Roger J., Fawcett, James W., Rogers, John H.
Publicado 2011

Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases

por Daboussi, Fayza, Zaslavskiy, Mikhail, Poirot, Laurent, Loperfido, Mariana, Gouble, Agnès, Guyot, Valerie, Leduc, Sophie, Galetto, Roman, Grizot, Sylvestre, Oficjalska, Danusia, Perez, Christophe, Delacôte, Fabien, Dupuy, Aurélie, Chion-Sotinel, Isabelle, Le Clerre, Diane, Lebuhotel, Céline, Danos, Olivier, Lemaire, Frédéric, Oussedik, Kahina, Cédrone, Frédéric, Epinat, Jean-Charles, Smith, Julianne, Yáñez-Muñoz, Rafael J., Dickson, George, Popplewell, Linda, Koo, Taeyoung, VandenDriessche, Thierry, Chuah, Marinee K., Duclert, Aymeric, Duchateau, Philippe, Pâques, Frédéric
Publicado 2012