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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

Javier del Rey Azpiri

Javier del Rey Azpiri

Research technician
Gene Therapy at Nervous System
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Jon Esandi Jauregui

Jon Esandi Jauregui

Predoctoral researcher
Gene Therapy at Nervous System
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Jorge Lunar Roldarn

Jorge Lunar Roldarn

Research technician
Gene Therapy at Nervous System
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Laia Rubio Ortega

Laia Rubio Ortega

Research technician
Gene Therapy at Nervous System
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Laura Rodriguez Estévez

Laura Rodriguez Estévez

Postdoctoral researcher
Gene Therapy at Nervous System
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Lluc Tarruell Roma

Lluc Tarruell Roma

Research technician
Gene Therapy at Nervous System
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Javier del Rey Azpiri

Javier del Rey Azpiri

Research technician
Gene Therapy at Nervous System
Read more
Jon Esandi Jauregui

Jon Esandi Jauregui

Predoctoral researcher
Gene Therapy at Nervous System
Read more
Jorge Lunar Roldarn

Jorge Lunar Roldarn

Research technician
Gene Therapy at Nervous System
Read more
Laia Rubio Ortega

Laia Rubio Ortega

Research technician
Gene Therapy at Nervous System
Read more
Laura Rodriguez Estévez

Laura Rodriguez Estévez

Postdoctoral researcher
Gene Therapy at Nervous System
Read more
Lluc Tarruell Roma

Lluc Tarruell Roma

Research technician
Gene Therapy at Nervous System
Read more

Publications

Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery.

PMID: 34857831
Journal: Scientific Reports
Year: 2021
Reference: Sci Rep. 2021 Dec 2;11(1):23358. doi: 10.1038/s41598-021-02694-1.
Impact factor: 4.38
Publication type: Paper in international publication
Authors: Kagiava, A, Richter, J, Tryfonos, C, Leal-Julia, M, Sargiannidou, I, Christodoulou, C, Bosch, A, Kleopa, K A et al.
DOI: 10.1038/s41598-021-02694-1

PolyPurine Reverse Hoogsteen Hairpins Work as RNA Species for Gene Silencing.

PMID: 34576188
Journal: INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Year: 2021
Reference: Int J Mol Sci. 2021 Sep 16;22(18). pii: ijms221810025. doi: 10.3390/ijms221810025.
Impact factor: 5.924
Publication type: Paper in international publication
Authors: Aubets, Eva, Chillon, Miguel, Ciudad, Carlos J, Noe, Veronique et al.
DOI: 10.3390/ijms221810025

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: 7.62
Publication type: Paper in international publication
Authors: Modol-Caballero, Guillem, Garcia-Lareu, Belen, Herrando-Grabulosa, Mireia, Verdes, Sergi, Lopez-Vales, Ruben, Pages, Gemma, Chillon, Miguel, Navarro, Xavier, Bosch, Assumpcio 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: 5.719
Publication type: Paper in international publication
Authors: Vogel-Gonzalez, Marina, Tallo-Parra, Marc, Herrera-Fernandez, Victor, Perez-Vilaro, Gemma, Chillon, Miguel, Nogues, Xavier, Gomez-Zorrilla, Silvia, Lopez-Montesinos, Inmaculada, Arnau-Barres, Isabel, Sorli-Redo, Maria Luisa et al.
DOI: 10.3390/nu13020562

Secreted and Transmembrane alphaKlotho Isoforms Have Different Spatio-Temporal Profiles in the Brain during Aging and Alzheimer's Disease Progression.

PMID: 26599613
Journal: PLoS One
Year: 2015
Reference: PLoS One. 2015 Nov 24;10(11):e0143623. doi: 10.1371/journal.pone.0143623. eCollection 2015.
Impact factor: 3.234
Publication type: Paper in international publication
Authors: Masso, Anna, Lizcano, Jose Miguel, Canete, Manuel, Garcia, Belen, Torres-Lista, Virginia, Puig, Meritxell, Bosch, Assumpcio, Chillon, Miguel, Gimenez-Llort, Lydia, Sanchez, Angela et al.
DOI: 10.1371/journal.pone.0143623

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

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: 0
Publication type: Paper in international publication
Authors: Homs, Judit, Pages, Gemma, Ariza, Lorena, Casas, Caty, Chillon, Miguel, Navarro, Xavier, Bosch, Assumpcio et al.
DOI: 10.1038/mtm.2013.7

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

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: 0
Publication type: Paper in international publication
Authors: Penalva, Cristina, Monfar, Merce, Chillon, Miguel, Piedra, Jose, Ontiveros, Maria, Miravet, Susana et al.
DOI: 10.1007/978-1-62703-679-5_12

Production of chimeric adenovirus.

PMID: 24132489
Journal: Methods in molecular biology (Clifton, N.J.)
Year: 2014
Reference: Methods Mol Biol. 2014;1089:231-43. doi: 10.1007/978-1-62703-679-5_16.
Impact factor: 0
Publication type: Paper in international publication
Authors: Miralles, Marta, Garcia, Marc, Tejero, Marcos, Bosch, Assumpcio, Chillon, Miguel et al.
DOI: 10.1007/978-1-62703-679-5_16

Short-fiber protein of ad40 confers enteric tropism and protection against acidic gastrointestinal conditions.

PMID: 23746215
Journal: Human Gene Therapy Methods
Year: 2013
Reference: Hum Gene Ther Methods. 2013 Aug;24(4):195-204. doi: 10.1089/hgtb.2012.096. Epub 2013 Jun 27.
Impact factor: 0
Publication type: Paper in international publication
Authors: Fernandez, Ester, Rodriguez, Ester, Romero, Carolina, Rio, Adolfo, Miralles, Marta, Raventos, Aida, Planells, Laura, Burgueno, Joan F, Hamada, Hirofumi, Perales, Jose Carlos et al.
DOI: 10.1089/hgtb.2012.096

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

Blog

News

The treatment, licensed to Klotho Neurosciences, is based on a technology developed at the Universitat Autònoma de Barcelona (UAB) with the participation of VHIR.

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.

Rates

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