Current Progress in Therapeutic Gene Editing for Monogenic Diseases von Prakash, Versha, Moore, Marc, Yáñez-Muñoz, Rafael J

Volltext

Comprehensive genomic access to vector integration in clinical gene therapy von Arens, Anne, Naldini, Luigi, Cattoglio, Claudia, Eckenberg, Ralph, Deichmann, Annette, Bartholomae, Cynthia C, Faber, Katrin, Nowrouzi, Ali, Paruzynski, Anna, Montini, Eugenio, Thrasher, Adrian J, Ali, Robin R, Saurin, William, Kirsten, Romy, Schwarzwaelder, Kerstin, Schmidt, Manfred, Balaggan, Kamaljit S, Glimm, Hanno, Gaspar, H Bobby, Cesana, Daniela, Biasco, Luca, Aiuti, Alessandro, Howe, Steven J, Recchia, Alessandra, Gabriel, Richard, Cohen-Haguenauer, Odile, Wang, Wei, Mavilio, Fulvio, Yáñez-Muñoz, Rafael J, von Kalle, Christof, Ball, Claudia R

Volltext

Correction: Andrew C. G. Porter (1955–2023) von Yáñez-Muñoz, Rafael J., Itzhaki, Jane E.

Volltext

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

Volltext

Andrew C. G. Porter (1955–2023) von Yáñez-Muñoz, Rafael J., Itzhaki, Jane E.

Volltext

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

Volltext

Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in von Popplewell, Linda, Koo, Taeyoung, Leclerc, Xavier, Duclert, Aymeric, Mamchaoui, Kamel, Gouble, Agnés, Mouly, Vincent, Voit, Thomas, Pâques, Frédéric, Cédrone, Frédéric, Isman, Olga, Yáñez-Muñoz, Rafael J, Dickson, George

Volltext

Integration-deficient Lentiviral Vectors: A Slow Coming of Age von Wanisch, Klaus, Yáñez-Muñoz, Rafael J

Volltext

Clinical applications of gene therapy for rare diseases: A review von Papaioannou, Ioannis, Owen, James S., Yáñez‐Muñoz, Rafael J.

Volltext

The British Society for Gene and Cell Therapy von Griesenbach, Uta, Yáñez-Muñoz, Rafael J

Volltext

Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models von Chilcott, Ellie M., Muiruri, Evalyne W., Hirst, Theodore C., Yáñez-Muñoz, Rafael J.

Volltext

Accumulation of the inhibitory receptor EphA4 may prevent regeneration of corticospinal tract axons following lesion von Fabes, Jez, Anderson, Patrick, Yáñez-Muñoz, Rafael J., Thrasher, Adrian, Brennan, Caroline, Bolsover, Stephen

Volltext

Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo von Nafchi, Neda A. M., Chilcott, Ellie M., Brown, Sharon, Fuller, Heidi R., Bowerman, Melissa, Yáñez-Muñoz, Rafael J.

Volltext

CAR-T in the clinic: drive with care von Yáñez-Muñoz, Rafael J., Grupp, Stephan A.

Volltext

Large-scale chondroitin sulfate proteoglycan digestion with chondroitinase gene therapy leads to reduced pathology and modulates macrophage phenotype following spinal cord contusio... von Bartus, Katalin, James, Nicholas D, Didangelos, Athanasios, Bosch, Karen D, Verhaagen, Joost, Yáñez-Muñoz, Rafael J, Rogers, John H, Schneider, Bernard L, Muir, Elizabeth M, Bradbury, Elizabeth J

Volltext

Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice von Ahlskog, Nina, Hayler, Daniel, Krueger, Anja, Kubinski, Sabrina, Claus, Peter, Hammond, Suzan M., Wood, Matthew J. A., Yáñez-Muñoz, Rafael J., Bowerman, Melissa

Volltext

Therapeutic strategies for spinal muscular atrophy: SMN and beyond von Bowerman, Melissa, Becker, Catherina G, Yáñez-Muñoz, Rafael J, Ning, Ke, Wood, Matthew J A, Gillingwater, Thomas H, Talbot, Kevin

Volltext

African Swine Fever Virus Protein pE296R Is a DNA Repair Apurinic/Apyrimidinic Endonuclease Required for Virus Growth in Swine Macrophages von REDREJO-RODRIGUEZ, Modesto, GARCFA-ESCUDERO, Ramon, YANEZ-MUNOZ, Rafael J, SALAS, Maria L, SALAS, José

Volltext

Effective gene therapy with nonintegrating lentiviral vectors von Yáñez-Muñoz, Rafael J, Ali, Robin R, Balaggan, Kamaljit S, MacNeil, Angus, Howe, Steven J, Schmidt, Manfred, Smith, Alexander J, Buch, Prateek, MacLaren, Robert E, Anderson, Patrick N, Barker, Susie E, Duran, Yanai, Bartholomae, Cynthia, von Kalle, Christof, Heckenlively, John R, Kinnon, Christine, Thrasher, Adrian J

Volltext

A study to assess a novel automated electrocardiogram technology in screening for atrial fibrillation von Sabar, Muhammad I., Ara, Farhana, Henderson, Alice, Ahmed, Omar, Potter, Christina, John, Isaac, Mitchell, Andrew R.J., Yáñez‐Muñoz, Rafael J., Kaba, Riyaz A.

Volltext