A. Brao, A. Sánchez, I. Rodríguez J. del Rey,Silvia Lope-Piedrafita, E. Prat, V. Nunes, M. Chillón, R. Estévez, and A. Bosch.
Gene therapy rescues brain edema and motor function in a mouse model of megalencephalic leukoencephalopathy with subcortical cysts
Molecular Therapy. (2025). 33(4):1434-1448.
DOI: 10.1016/j.ymthe.2025.02.046
IF: 12.0
RESEARCH GROUPS
Gene Therapy for Nervous System
3
Number of publications
100.00
%
% Q1
66.67
%
% D1
100.00
%
% Leadership
66.67
%
% Female Leadership
1.95
CROWN (2021-2025)
22.73
%
HCP10% (2021-2025)
100.00
%
% Open access
33.33
%
% International Collaboration
1
Highly cited papers / HCP 1% (2021-2025)
1
Publications in high-IF journals with high Altmetric scores
7
Number of Ongoing Projectes (total)
0
International ongoing projects
1
Number of New projects (total)
0
International new projects
0
Number of Active clinical Trials
0
Number of New Clinical Trials
1
Number of Theses
In 2025, the group advanced gene therapy strategies for neurological and age-related diseases, focusing on AAV vector development and CNS-targeted applications. They contributed to projects such as TERAV+ and launched initiatives on chronokines and Klotho-based therapies for neurodegeneration and aging. Preclinical studies showed therapeutic efficacy, including improvements in cognitive deficits, brain edema, motor function, and longevity, with results published in three first-decile journals. The group also advanced the characterization of Klotho in the CNS and its role in Alzheimer’s disease. Notably, an AAV-based gene therapy for ALS developed at UAB received FDA Orphan Drug Designation. The group also contacted AEMPS to request a clinical trial for gene therapy targeting the ultra-rare pediatric disease SPG52. The UPV service increased productivity (~250 viral batches/year), renewed ISO9001:2015 certification, and was reaccredited as a TECNIO service for its high-quality standards.
CORE/MISSIONS
- Innovative & Advanced Therapies
- Rare Diseases
PROGRAMS
TEAM
Group Leader
Miguel Chillón Rodriguez
Principal Investigator (PI)
Miguel Chillón Rodriguez
Researchers
Joan Roig Soriano, Beatriz Almolda Ardid, Laura Rodriguez Estevez, Susana Miravet Delgado, Carmen Plasencia Castillo
PhD Students
David Ramírez Gómez, Anna Behr, Chiara Piccinini, Oriol Verdu Bautista
Lab Technicians
Jorge Lunar Roldan, Javier del Rey Azpiri, Laia Rubio Ortega, Israel Alcazar Corrales, Elsa Ibarrola Carrasco, Miriam Calles Bastande, Nerea Jimenez Hernandez, Jose Piedra Miravet, Sara Rodriguez Navas
SELECTED PUBLICATIONS
Roig-Soriano J, Edo A, Verdés S, Martín-Alonso C, Sánchez-de-Diego C, Rodriguez-Estevez L, Serrano AL, Abraham CR, Bosch A, Ventura F, Jordan BA, Muñoz-Cánoves P; Chillón M.
Long-term effects of s-KL treatment in wild-type mice, enhancing longevity, physical well-being, and neurological resilience.
Molecular Therapy. (2025) 33(4):1449-1465.
DOI: 10.1016/j.ymthe.2025.02.030
IF: 12.0
J. Esandi; P. Renault; M. Dolores Capilla-López; R. Blanch; A. Edo; D. Ramirez-Gomez; A. Bosch; B. Almolda; C.A. Saura; J. Giraldo; M. Chillón.
HEBE: a novel chimeric chronokine for ameliorating memory deficits in Alzheimer’s disease.
Biomedicine and Pharmacotherapy. (2025). Feb:183:117815.
DOI: 10.1016/j.biopha.2025.117815
IF: 7.5
SELECTED PROJECTS
TERAV+
Principal Investigator: Coordinador:Manel Joan (IDIBAPS). IP(VHIR): Miguel Chillon
Agency: ISCIII. REDES DE INVESTIGACIÓN COOPERATIVA ORIENTADAS A RESULTADOS EN SALUD (RICORS). RD24/0014/0039
Funding: 140,239€
Period: 01/01/25 to 31/12/27
Towards an effective and safe therapy for hormonal-deficiency osteoporosis based on klotho protein
Principal Investigator: Joan Roig
Agency: CaixaImpulse Innovation 2024. La Caixa. (CI24-20581)
Funding: 150,000€
Period: 15/10/24 to 14/10/26
The European Rare Diseases Research Alliance (ERDERA)
Principal Investigator: MIguel Chillón
Agency: Horizon Europe (UE)
Funding: 125,755€
Period: 01/09/24 to 31/08/28
Study and development of the first technological platform to assay adeno-associated viruses for gene therapy treatments
Principal Investigator: MIguel Chillon
Agency: ISCIII y CDTI. CONVOCATORIA proyectos de i+d+i vinculados a la medicina personalizada y terapias avanzadas. PMPTA22/00048
Funding: 296,571€
Period: 01/01/23 to 30/06/25
Gene Therapy for GNB1 Encephalopathy
Principal Investigator: Miguel Chillón
Agency: Asociaciones de Pacientes: Gene Rebelde y GNB1 España
Funding: 450,000€
Period: 11/2020 to 12/2028
PATENTS
Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
Priority Number: US63/330.684.
Priority Date: 13/04/2022
Applicants: UAB (72.5%); ICREA (5%); VHIR (2.5%); CIBERNED (20%). Licensing company: Klotho Neurosciences (USA).
Secreted splicing variant of mammal Klotho as a medicament for cognition and behaviour impairments
Priority Number: EP15195470.8.
Priority Date: 19/11/2015
Applicants: UAB (66.25%), ICREA (22.5%), VHIR (11.25%). Licencing company: Klotho Neurosciences (USA)
Secreted Splicing variant of klotho for treating bone disorders
Priority Number: WO2022/243519-A1.
Priority Date: 21/5/2021
Applicants: UAB (57.5%), ICREA (15%), VHIR (7.5%), UB (20%)
Secreted splicing variant of klotho for extending lifespan
Priority Number: EP22383171.0.
Priority Date: 2/12/2022
Applicants: UAB (70%), ICREA (20%), VHIR (10%)
Secreted splicing variant of klotho for treating muscle disorders
Priority Number: EP22383173.6.
Priority Date: 2/12/2022
Applicants: UAB (62.5%), ICREA (21%), VHIR (9.25%), UP(7.5%)
DISSEMINATION ACTIVITIES
Estem preparats per viure més del que ens pertoca biològicament?
Place: Radio Program "El pla B". RNE
Date: 03/06/2025
Brief: Interview in a radio program about our research on healthy aging
Terapia Génica para SPG52
Place: Barcelona
Date: 26/2/2025
Brief: Meeting with families and patients of the Spanish Association of Familial Paraparesis, and subsequent conference
Terapia Génica para enfermedades raras
Place: Valencia
Date: 28/2/2025
Brief: Meeting and subsequent conference with researchers, medical students and patients of various rare diseases, in a day organized by the Cardenal Herrera University and the ANTIAN association of ADSL patients.
Cronoquinas: envejecimiento saludable y longevidad. Klotho como paradigma
Place: Barcelona
Date: 9/5/2025
Brief: Participation (invited speaker) in the Senescence and Aging Symposium. Dr. Antoni Esteve i Subirana Chair of Research in Pharmacology, University of Barcelona, open to the general public
