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Gene Therapy at Nervous System

Medicine still has many challenges to solve specially on complex diseases were a large number of both, genetic and environmental factors are involved. Among them, neurodegenerative and autoimmune disorders affecting the central nervous system have attracted attention because CNS is difficult to access and to manipulate with classical pharmacological treatments and, in addition, there are no effective curative treatments for these diseases. To address these problems, we have focused our research interests in gene therapy strategies for autoimmune diseases and neurodegenerative disorders specially those associated to aging.
 

Team

Maria Pallares Masmitja

Maria Pallares Masmitja

Postdoctoral researcher
Gene Therapy at Nervous System
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Miriam Calles Bastande

Miriam Calles Bastande

Research assistant
Gene Therapy at Nervous System
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Rebeca Blanch Garcia

Rebeca Blanch Garcia

Research technician
Gene Therapy at Nervous System
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Andrea Onieva Salgado

Andrea Onieva Salgado

Predoctoral researcher
Gene Therapy at Nervous System
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Ángela Sánchez Osuna

Ángela Sánchez Osuna

Postdoctoral researcher
Gene Therapy at Nervous System
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Elsa Ibarrola Carrasco

Elsa Ibarrola Carrasco

Research technician
Gene Therapy at Nervous System
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Maria Pallares Masmitja

Maria Pallares Masmitja

Postdoctoral researcher
Gene Therapy at Nervous System
Read more
Miriam Calles Bastande

Miriam Calles Bastande

Research assistant
Gene Therapy at Nervous System
Read more
Rebeca Blanch Garcia

Rebeca Blanch Garcia

Research technician
Gene Therapy at Nervous System
Read more
Andrea Onieva Salgado

Andrea Onieva Salgado

Predoctoral researcher
Gene Therapy at Nervous System
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Ángela Sánchez Osuna

Ángela Sánchez Osuna

Postdoctoral researcher
Gene Therapy at Nervous System
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Elsa Ibarrola Carrasco

Elsa Ibarrola Carrasco

Research technician
Gene Therapy at Nervous System
Read more

Publications

AAV-mediated expression of secreted and transmembrane alphaKlotho isoforms rescues relevant aging hallmarks in senescent SAMP8 mice.

PMID: 35274439
Journal: AGING CELL
Year: 2022
Reference: Aging Cell. 2022 Apr;21(4):e13581. doi: 10.1111/acel.13581. Epub 2022 Mar 10.
Impact factor: 9.304
Publication type: Paper in international publication
Authors: Bosch, A, Pallas, M, Chillon, Miguel, Abraham, C R, Espinosa-Parrilla, J F, Roig-Soriano, J, Grinan-Ferre, C et al.
DOI: 10.1111/acel.13581

PROTEOMIC QUANTITATIVE STUDY OF DORSAL ROOT GANGLIA AND SCIATIC NERVE IN TYPE 2 DIABETIC MICE.

PMID: 34856394
Journal: Molecular Metabolism
Year: 2022
Reference: Mol Metab. 2022 Jan;55:101408. doi: 10.1016/j.molmet.2021.101408. Epub 2021 Nov 30.
Impact factor: 7.422
Publication type: Paper in international publication
Authors: Leal-Julia, Marc, Vilches, Jorge J, Onieva, Andrea, Verdes, Sergi, Sanchez, Angela, Chillon, Miguel, Navarro, Xavier, Bosch, Assumpcio et al.
DOI: 10.1016/j.molmet.2021.101408

Specific Expression of Glial-Derived Neurotrophic Factor in Muscles as Gene Therapy Strategy for Amyotrophic Lateral Sclerosis.

PMID: 33786805
Journal: Neurotherapeutics
Year: 2021
Reference: Neurotherapeutics. 2021 Apr;18(2):1113-1126. doi: 10.1007/s13311-021-01025-6. Epub 2021 Mar 30.
Impact factor:
Publication type: Paper in international publication
Authors: Bosch, Assumpcio; Chillon, Miguel; Garcia-Lareu, Belen; Herrando-Grabulosa, Mireia; Lopez-Vales, Ruben; Modol-Caballero, Guillem; Navarro, Xavier; Pages, Gemma; Verdes, Sergi et al.
DOI: 10.1007/s13311-021-01025-6

Low Zinc Levels at Admission Associates with Poor Clinical Outcomes in SARS-CoV-2 Infection.

PMID: 33572045
Journal: Nutrients
Year: 2021
Reference: Nutrients. 2021 Feb 9;13(2). pii: nu13020562. doi: 10.3390/nu13020562.
Impact factor:
Publication type: Paper in international publication
Authors: Arnau-Barres, Isabel; Chillon, Miguel; Diez, Juana; Garcia-Giralt, Natalia; Gomez-Zorrilla, Silvia; Guerri-Fernandez, Robert; Herrera-Fernandez, Victor; Horcajada, Juan Pablo; Lopez-Montesinos, Inmaculada; Nogues, Xavier et al.
DOI: 10.3390/nu13020562

The pleiotropic neuroprotective effects of resveratrol in cognitive decline and Alzheimer's disease pathology: from antioxidant to epigenetic therapy.

PMID: 33571701
Journal: AGEING RESEARCH REVIEWS
Year: 2021
Reference: Ageing Res Rev. 2021 May;67:101271. doi: 10.1016/j.arr.2021.101271. Epub 2021 Feb 8.
Impact factor:
Publication type: Review in international publication
Authors: Andres-Lacueva, Cristina; Bellver-Sanchis, Aina; Chillon, Miguel; Corpas, Ruben; Grinan-Ferre, Christian; Izquierdo, Vanessa; Pallas, Merce; Roig-Soriano, Joan; Sanfeliu, Coral; Somogyvari, Milan et al.
DOI: 10.1016/j.arr.2021.101271

A Transgenic Model Reveals the Role of Klotho in Pancreatic Cancer Development and Paves the Way for New Klotho-Based Therapy.

PMID: 34944918
Journal: Cancers
Year: 2021
Reference: Cancers (Basel). 2021 Dec 15;13(24). pii: cancers13246297. doi: 10.3390/cancers13246297.
Impact factor: 6.639
Publication type: Paper in international publication
Authors: Arbel Rubinstein, Tammi, Reuveni, Inbal, Hesin, Arkadi, Klein-Goldberg, Anat, Olauson, Hannes, Larsson, Tobias E, Abraham, Carmela R, Zeldich, Ella, Bosch, Assumpcio, Chillon, Miguel et al.
DOI: 10.3390/cancers13246297

Megalencephalic Leukoencephalopathy: Insights Into Pathophysiology and Perspectives for Therapy.

PMID: 33551753
Journal: Frontiers in Cellular Neuroscience
Year: 2020
Reference: Front Cell Neurosci. 2021 Jan 22;14:627887. doi: 10.3389/fncel.2020.627887. eCollection 2020.
Impact factor: 3.921
Publication type: Review in international publication
Authors: Bosch, Assumpcio, Estevez, Raul et al.
DOI: 10.3389/fncel.2020.627887

Cerebellar Astrocyte Transduction as Gene Therapy for Megalencephalic Leukoencephalopathy.

PMID: 32372403
Journal: Neurotherapeutics
Year: 2020
Reference: Neurotherapeutics. 2020 Oct;17(4):2041-2053. doi: 10.1007/s13311-020-00865-y.
Impact factor: 6.035
Publication type: Paper in international publication
Authors: Bosch, Assumpcio, Estevez, Raul, Nunes, Virginia, Sanchez, Angela, Garcia-Lareu, Belen, Puig, Meritxell, Prat, Esther, Ruberte, Jesus, Chillon, Miguel et al.
DOI: 10.1007/s13311-020-00865-y

Gene therapy for overexpressing Neuregulin 1 type I in skeletal muscles promotes functional improvement in the SOD1(G93A) ALS mice.

PMID: 32032731
Journal: NEUROBIOLOGY OF DISEASE
Year: 2020
Reference: Neurobiol Dis. 2020 Apr;137:104793. doi: 10.1016/j.nbd.2020.104793. Epub 2020 Feb 4.
Impact factor: 5.332
Publication type: Paper in international publication
Authors: Herrando-Grabulosa, Mireia, Garcia-Lareu, Belen, Solanes, Neus, Verdes, Sergi, Osta, Rosario, Francos-Quijorna, Isaac, Lopez-Vales, Ruben, Calvo, Ana Cristina, Bosch, Assumpcio, Navarro, Xavier et al.
DOI: 10.1016/j.nbd.2020.104793

Clinical and Histopathological Amelioration of Experimental Autoimmune Encephalomyelitis by AAV Vectors Expressing a Soluble Interleukin-23 Receptor.

PMID: 28593439
Journal: Neurotherapeutics
Year: 2017
Reference: Neurotherapeutics. 2017 Oct;14(4):1095-1106. doi: 10.1007/s13311-017-0545-8.
Impact factor: 5.166
Publication type: Paper in international publication
Authors: Eixarch, Herena, Tejero, Marcos, Costa, Carme, Hirota, Keiji, Castano, A Raul, Puig, Meritxell, Stockinger, Gitta, Montalban, Xavier, Bosch, Assumpcio, Espejo, Carmen et al.
DOI: 10.1007/s13311-017-0545-8

Mechanics of Viral Chromatin Reveals the Pressurization of Human Adenovirus.

PMID: 26491879
Journal: ACS NANO
Year: 2015
Reference: ACS Nano. 2015 Nov 24;9(11):10826-33. doi: 10.1021/acsnano.5b03417. Epub 2015 Oct 28.
Impact factor:
Publication type: Paper in international publication
Authors: Chillon, Miguel; Condezo, Gabriela N; de Pablo, Pedro J; Flint, S Jane; Ortega-Esteban, Alvaro; Perez-Berna, Ana J; Reguera, David; San Martin, Carmen et al.
DOI: 10.1021/acsnano.5b03417

Development of a rapid, robust, and universal picogreen-based method to titer adeno-associated vectors.

PMID: 25640021
Journal: Human Gene Therapy Methods
Year: 2015
Reference: Hum Gene Ther Methods. 2015 Feb;26(1):35-42. doi: 10.1089/hgtb.2014.120.
Impact factor: 2.436
Publication type: Paper in international publication
Authors: Penalva, Cristina, Monfar, Merce, Chillon, Miguel, Miravet, Susana, Ontiveros, Maria, Piedra, Jose et al.
DOI: 10.1089/hgtb.2014.120

Mechanics of Viral Chromatin Reveals the Pressurization of Human Adenovirus.

PMID: 26491879
Journal: ACS Nano
Year: 2015
Reference: ACS Nano. 2015 Nov 24;9(11):10826-33. doi: 10.1021/acsnano.5b03417. Epub 2015 Oct 28.
Impact factor: 12.881
Publication type: Paper in international publication
Authors: Ortega-Esteban, Alvaro, Condezo, Gabriela N, Perez-Berna, Ana J, Chillon, Miguel, Flint, S Jane, Reguera, David, San Martin, Carmen, de Pablo, Pedro J et al.
DOI: 10.1021/acsnano.5b03417

Structures of Adenovirus Incomplete Particles Clarify Capsid Architecture and Show Maturation Changes of Packaging Protein L1 52/55k.

PMID: 26178997
Journal: JOURNAL OF VIROLOGY
Year: 2015
Reference: J Virol. 2015 Sep;89(18):9653-64. doi: 10.1128/JVI.01453-15. Epub 2015 Jul 15.
Impact factor: 4.439
Publication type: Paper in international publication
Authors: San Martin, Carmen, Condezo, Gabriela N, Marabini, Roberto, Ayora, Silvia, Carazo, Jose M, Alba, Raul, Chillon, Miguel et al.
DOI: 10.1128/JVI.01453-15

Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy.

PMID: 26015946
Journal: Molecular therapy. Methods & clinical developm
Year: 2014
Reference: Mol Ther Methods Clin Dev. 2014 Jan 15;1:7. doi: 10.1038/mtm.2013.7. eCollection 2014.
Impact factor:
Publication type: Paper in international publication
Authors: Ariza, Lorena; Bosch, Assumpcio; Casas, Caty; Chillon, Miguel; Homs, Judit; Navarro, Xavier; Pages, Gemma et al.
DOI: 10.1038/mtm.2013.7

Construction, production, and purification of recombinant adenovirus vectors.

PMID: 24132485
Journal: Methods in molecular biology (Clifton, N.J.)
Year: 2014
Reference: Methods Mol Biol. 2014;1089:159-73. doi: 10.1007/978-1-62703-679-5_12.
Impact factor:
Publication type: Paper in international publication
Authors: Chillon, Miguel; Miravet, Susana; Monfar, Merce; Ontiveros, Maria; Penalva, Cristina; Piedra, Jose et al.
DOI: 10.1007/978-1-62703-679-5_12

Enhancing the Biological Relevance of Secretome-Based Proteomics by Linking Tumor Cell Proliferation and Protein Secretion.

PMID: 24897304
Journal: J PROTEOME RES
Year: 2014
Reference: J Proteome Res. 2014 Aug 1;13(8):3706-3721. doi: 10.1021/pr500304g. Epub 2014 Jul 15.
Impact factor: 5.001
Publication type: Paper in international publication
Authors: Gregori, Josep, Mendez, Olga, Katsila, Theodora, Pujals, Mireia, Salvans, Candida, Villarreal, Laura, Sanchez, Alex, Villanueva, Josep, Tabernero, Josep, Arribas, Joaquin et al.
DOI: 10.1021/pr500304g

Central nervous system delivery of helper-dependent canine adenovirus corrects neuropathology and behavior in mucopolysaccharidosis type VII mice.

PMID: 24299455
Journal: HUM GENE THER
Year: 2014
Reference: Hum Gene Ther. 2014 Mar;25(3):199-211. doi: 10.1089/hum.2013.152. Epub 2014 Jan 23.
Impact factor: 3.623
Publication type: Paper in international publication
Authors: Ariza, Lorena, Gimenez-Llort, Lydia, Pages, Gemma, Garcia-Lareu, Belen, Serratrice, Nicolas, Cots, Dan, Thwaite, Rosemary, Chillon, Miguel, Kremer, Eric J, Bosch, Assumpcio et al.
DOI: 10.1089/hum.2013.152

Differentiated neuroprogenitor cells incubated with human or canine adenovirus, or lentiviral vectors have distinct transcriptome profiles.

PMID: 23922808
Journal: PLoS One
Year: 2013
Reference: PLoS One. 2013 Jul 26;8(7):e69808. doi: 10.1371/journal.pone.0069808. Print 2013.
Impact factor:
Publication type: Paper in international publication
Authors: Astrologo, Letizia; Chillon, Miguel; Costa, Rossella; Gennetier, Aurelie; Ibanes, Sandy; Kremer, Eric J; Licursi, Valerio; Negri, Rodolfo; Piersanti, Stefania; Roncaglia, Enrica et al.
DOI: 10.1371/journal.pone.0069808

Functional characterization of the human mariner transposon Hsmar2.

PMID: 24039890
Journal: PLoS One
Year: 2013
Reference: PLoS One. 2013 Sep 11;8(9):e73227. doi: 10.1371/journal.pone.0073227. eCollection 2013.
Impact factor: 3.73
Publication type: Paper in international publication
Authors: Gil, Estel, Bosch, Assumpcio, Lampe, David, Lizcano, Jose M, Perales, Jose C, Danos, Olivier, Chillon, Miguel et al.
DOI: 10.1371/journal.pone.0073227

Differentiated neuroprogenitor cells incubated with human or canine adenovirus, or lentiviral vectors have distinct transcriptome profiles.

PMID: 23922808
Journal: PLoS One
Year: 2013
Reference: PLoS One. 2013 Jul 26;8(7):e69808. doi: 10.1371/journal.pone.0069808. Print 2013.
Impact factor: 3.73
Publication type: Paper in international publication
Authors: Licursi, Valerio, Costa, Rossella, Roncaglia, Enrica, Gennetier, Aurelie, Ibanes, Sandy, Chillon, Miguel, Negri, Rodolfo, Tagliafico, Enrico, Kremer, Eric J, Saggio, Isabella et al.
DOI: 10.1371/journal.pone.0069808

Adeno-associated virus enhances wild-type and oncolytic adenovirus spread.

PMID: 24020980
Journal: Human Gene Therapy Methods
Year: 2013
Reference: Hum Gene Ther Methods. 2013 Dec;24(6):372-80. doi: 10.1089/hgtb.2013.124. Epub 2013 Oct 8.
Impact factor: 0
Publication type: Paper in international publication
Authors: Laborda, Eduardo, Puig-Saus, Cristina, Cascallo, Manel, Chillon, Miguel, Alemany, Ramon et al.
DOI: 10.1089/hgtb.2013.124

Blog

News

The European Rare Diseases Research Alliance (ERDERA) kicked off this September, with an estimated budget of 380 million euros and the aim of improving the lives of 30 million rare disease patients in Europe and beyond.

This gene therapy protects mice against cognitive deficits associated with aging, improves motor function, and delays the onset of diseases such as amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease.

Over the course of two days, experts presented the latest advances in vectors, different gene modification techniques and their transfer to clinical practice.

Rates

Check the current rates for the services offered by the Gene Therapy at Nervous System research group.