Gene Therapy for Nervous System – VHIR Annual Report 2024

Publications

75.00

%Q1

Number of citations

100.00

% Open Access Publications

Ongoing Projects

Number of Presented Theses

Number of Active Clinical Trials

Our research group is focused on the study of the molecular mechanisms involved in genetic diseases that cause nervous system dysfunction, with the final goal of developing effective and safe gene therapy strategies for those diseases. By applying state-of-the-art techniques such as 2D and 3D cultures of iPSC-derived neurons from patients, in vivo gene therapy strategies in animal models (including transgenic models), -omic analysis (transcriptomic and spatial transcriptomic analysis), novel viral vectors, etc, our laboratory has three major complementary strategic research lines: 1) Natural and chimeric chronokines for age-associated disorders 2) AAV gene therapy strategies for rare diseases affecting the CNS:

Spastic Paraplegia type 52 (SPG52)
GNB1 Encephalopathy
ADSL Deficiency
Megalencephalic Leukoencephalopathy with subcortical Cysts (MLC)
Amyotrophic lateral sclerosis (ALS)

3) Design, generation and production of AAV and adenovirus vectors

eCORE

Advanced Therapies and Advanced Interventions, Nanomedicine, Transplant & Donation
Chronic, Prevalent Diseases & Aging

Team

Group Leader
Miguel Chillon Rodriguez

Principal Investigator (PI)
Miguel Chillon Rodriguez

Researchers
Assumpció Bosch Merino, Joan Roig Soriano, Beatriz Almolda Ardid, Angela Sánchez Osuna, Sergi Verdes Franquesa

PhD Students
Laura Rodriguez Estevez, David Ramirez Gomez, Anna Behr, Chiara Piccinini, Alejandro Brao Gonzalez, Rubén Guerrero Yagüe, Maria Page Galocha, Irina Rodriguez Reverté, Judith Sauleda Sauleda, Oriol Verdú Bautista

Lab Technicians
Javier del Rey Azpiry, Jorge Lunar Roldan, Susana Miravet Delgado, Maria Ontiveros Gonzalez, Laia Rubio Ortega, Israel Alcazar Corrales, Elsa Ibarrola Carrasco, Miriam Calles Bastande, Nerea Jimenez Hernandez

Selected Publications

Ciudad CJ, Valiuska S, Rojas JM, Nogales-Altozano P, Aviñó A, Eritja R, Chillón M, Sevilla N, Noé V.
Polypurine Reverse Hoogsteen hairpins as a therapeutic tool for SARS-CoV-2 infection.
J Biol Chem. Oct 10:107884
DOI: 10.1016/j.jbc.2024.107884
IF: 4.0

Bunuales M, Garduno A, Chillon M, Bosch A, Gonzalez-Aparicio M, Espelosin M, Garcia-Gomara M, Rico A.J, Garcia-Osta A, Cuadrado-Tejedor M, Lanciego J.L, Hernandez-Alcoceba R.
Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles.
Gene Therapy. Sep;31(9-10):455-466
DOI: 10.1038/s41434-024-00466-w
IF: 4.6

10.1038/s41434-024-00466-w
Protocol for Generation of Semi-guided Cortical Organoids with Complex Neural Oscillations
Nature Protocols. Sep;19(9):2712-2738.
DOI: 10.1038/s41596-024-00994-0
IF: 13.1

Favaro MTP, Alamo P, Roher N, Chillon M, Lascorz J, Márquez M, Corchero JL, Mendoza R, Martínez-Torró C, Ferrer-Miralles N, Ferreira LCS, Mangues R, Vázquez E, Parladé E, Villaverde A.
Adjuvant-free microscale granules made of the SARS-CoV-2 spike protein trigger an anti-virus neutralizing response.
ACS Materials Letters, 6:954-962.
DOI: 10.1021/acsmaterialslett.3c01643
IF: 11.4

Selected Projects

Towards an effective and safe therapy for hormonal-deficiency osteoporosis based on klotho protein. CaixaImpulse Innovation 2024. La Caixa. (CI24-20581)
Principal Investigator: Joan Roig Soriano
Agency: La Caixa
Funding: 150,000 €
Period: 15/10/2024 - 14/10/2026

The European Rare Diseases Research Alliance (ERDERA)
Principal Investigator: Coordinator at VHIR (Mar Mañu); (PI of Gene Therapy: Miguel Chillon)
Agency: Horizon Europe. European Union
Funding: 125,755€
Period: 01/09/2024 - 31/08/2028

Estudio y desarrollo de la primera plataforma tecnológica de ensayos para virus adenoasociados en terápia génica. PMPTA22/00048
Principal Investigator: Miguel Chillon
Agency: ISCIII-CDTI.
Funding: 296,571 €
Period: 01/04/2023 - 31/03/2025

Therapeutical modulation of the microglia-neuronal interaction by chronokines to treat chronic brain inflammation associated with aging. (μ−4-WELL-AGING). (PID2022-142624OB-I00)
Principal Investigator: Miguel Chillon and Beatriz Almolda
Agency: Proyectos de Generación de Conocimiento. Ministerio de Ciencia e Innovación (MICINN).
Funding: 125,000 €
Period: 01/09/2023 - 31/08/2026

Gene therapy to treat Megalencephalic Leukodystrophy with subcortical cysts (MLC). (ELA 2022-00412)
Principal Investigator: Assumpció Bosch
Agency: European Leukodystrophy Association
Funding: 189,654 €
Period: 01/03/2023 - 28/02/2025

Patents

Nucleic acid constructs and vectors for podocyte specific expression
Priority Number: EP22382421.0.
Priority Date: 02/05/2022
Applicants: VHIR; Ninevah; UAB; ICREA

Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
Priority Number: US63/330.684.
Priority Date: 13/04/2022
Applicants: UAB (82.5%); CIBERNED (10%); ICREA (5%); VHIR (2,5%)

Selected Dissemination Activities

La raresa té dret a l'esperança. Per una teràpia gènica
Place: Centre Cultural Cambrils
Date: 01/01/1970
Brief: Conference on rare genetic diseases and how to treat them with advanced therapies