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Bueren, Juan
318  results:
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1

Lentivirus-mediated gene therapy corrects ribosomal biogene..:

Giménez, Yari ; Palacios, Manuel ; Sánchez-Domínguez, Rebeca...
JCI Insight.  9 (2024)  10 - p. , 2024
Link: https://doi.org/10.1172/..
 
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2

Lentiviral-Mediated Gene Therapy for Patients with Fanconi ..:

Czechowicz, Agnieszka ; Sevilla, Julian ; Booth, Claire...
Transplantation and Cellular Therapy.  29 (2023)  2 - p. S89 , 2023
Link: https://doi.org/10.1016/..
 
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3

Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Pati..:

Booth, Claire ; Sevilla, Julian ; Rao, Gayatri...
Transplantation and Cellular Therapy.  29 (2023)  2 - p. S2-S3 , 2023
Link: https://doi.org/10.1016/..
 
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4

The IRDiRC Chrysalis Task Force: making rare disease resear..:

Beaverson, Katherine L. ; Julkowska, Daria ; Letinturier, Mary Catherine V....
Therapeutic Advances in Rare Disease.  4 (2023)  - p. , 2023
Link: https://doi.org/10.1177/..
 
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5

Improved efficacy of mesenchymal stromal cells stably expre..:

Hervás-Salcedo, Rosario ; Fernández-García, María ; Hernando-Rodríguez, Miriam...
Frontiers in Immunology.  14 (2023)  - p. , 2023
Link: https://doi.org/10.3389/..
 
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6

Severe Leukocyte Adhesion Deficiency-I (LAD-I) Lentiviral-M..:

Booth, Claire ; Sevilla, Julian ; Lopez, Maria Chitty...
Clinical Immunology.  250 (2023)  - p. 109354 , 2023
Link: https://doi.org/10.1016/..
 
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7

Gene therapy restores the transcriptional program of hemato..:

Lasaga, Miren ; Río, Paula ; Vilas-Zornoza, Amaia...
Haematologica.  , 2023
Link: https://doi.org/10.3324/..
 
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8

Lentiviral-Mediated Gene Therapy for Severe Pyruvate Kinase..:

Shah, Ami J ; Lorenzo, José Luis López ; Sevilla, Julian...
Transplantation and Cellular Therapy.  29 (2023)  2 - p. S225-S226 , 2023
Link: https://doi.org/10.1016/..
 
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9

Access to gene therapy for rare diseases when commercializa..:

Fox, Thomas ; Bueren, Juan ; Candotti, Fabio...
Nature Medicine.  29 (2023)  3 - p. 518-519 , 2023
Link: https://doi.org/10.1038/..
 
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10

Specific correction of pyruvate kinase deficiency-causing p..:

Fañanas-Baquero, Sara ; Morín, Matías ; Fernández, Sergio...
Frontiers in Genome Editing.  5 (2023)  - p. , 2023
Link: https://doi.org/10.3389/..
 
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11

Role of Hospital Exemption in Europe: position paper from t..:

Sánchez-Guijo, Fermín ; Avendaño-Solá, Cristina ; Badimón, Lina...
Bone Marrow Transplantation.  58 (2023)  6 - p. 727-728 , 2023
Link: https://doi.org/10.1038/..
 
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12

Adenine base editing efficiently restores the function of F..:

Siegner, Sebastian M. ; Ugalde, Laura ; Clemens, Alexandra...
Nature Communications.  13 (2022)  1 - p. , 2022
Link: https://doi.org/10.1038/..
 
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13

Upregulation of NKG2D ligands impairs hematopoietic stem ce..:

Casado, José A. ; Valeri, Antonio ; Sanchez-Domínguez, Rebeca...
Journal of Clinical Investigation.  132 (2022)  15 - p. , 2022
Link: https://doi.org/10.1172/..
 
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14

CIBERER: Spanish national network for research on rare dise..:

Luque, Juan ; Mendes, Ingrid ; Gómez, Beatriz...
Clinical Genetics.  101 (2022)  5-6 - p. 481-493 , 2022
Link: https://doi.org/10.1111/..
 
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15

Preclinical safety and efficacy of lentiviral-mediated gene..:

Mesa-Núñez, Cristina ; Damián, Carlos ; Fernández-García, María...
Molecular Therapy - Methods & Clinical Development.  26 (2022)  - p. 459-470 , 2022
Link: https://doi.org/10.1016/..
 
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