GEN REBELDE FOUNDATION
HISTORIA Y MISION.
The microdonation initiative is carried out by Marc León, together with the patients' families, for a research project by Dr. Miguel Chillón in gene therapy. Half a year ago, Marc León launched a solidarity initiative on the internet, together with relatives of other Spanish patients, to help raise funds for the study of GNB1 encephalopathy in the Vall d'Genetic Therapy in the Nervous System research group. Hebron Research Institute (VHIR), led by Dr. Miguel Chillón. This pathology has no cure and there are not even exhaustively tested medications available to treat serious symptoms such as epilepsy. The long-term clinical prognosis is also not known, as studies began only a few years ago. So far, more than 53,000 euros have been raised in total, between the initial contribution from the company EPS Container Line, and the individual contributions obtained through the challenge on the MiGranoDeArena website. Thanks to these first donations, we will be able to better understand the disease, which is still largely unknown, due to the small number of patients who suffer from this ultra-rare genetic mutation (just over 100 cases diagnosed in the world). The challenge now is to obtain more funds to begin the second stage of the research, in which therapeutic AAV-GNB1 vectors will be generated, validate the expression of the therapeutic gene, and iPSCs (induced pluripotent stem cells) from two GNB1 patients will also be generated. and their progenitors, and their derivation to neurons.
PROYECTOS
ACTUALES.
Dr. M. Chillon created his research group in 2001, focusing from the beginning on four main objectives: (1) gene therapy for cognitive deficits associated with aging; (2) gene therapy for autoimmune diseases; (3) gene therapy for rare diseases that affect the nervous system and (4) development of more efficient gene therapy vectors. Dr. Chillon is the author of 76 scientific articles, 3 reviews, 5 book chapters, editor of 1 book (H index: 27; 3470 citations, Scopus), and has supervised 14 doctoral theses (4 in progress) and 22 undergraduate students. Master. In the last five years, Dr. Chillon has obtained different national and international projects such as European Joint Program-Rare Diseases AC20/00051; Society Challenges. PID2019-104034RB-I00; Collaboration Challenges RTC2019-006879-1; RICORS of ISCIII RD21/0017/0008; Santander Foundation (2020); CERVERA IDI-2020-0258; ALS International (2018-0512); SmartMoney-UAB (2017); and Marato TV3, 201607.10. Regarding knowledge transfer, Dr. Chillon's group has generated 10 patents and founded 2 spin-off companies: NanoTherapix (2009) for gene and cell therapy for diseases of the immune system; and Klogene (2016) for gene therapy of cognitive impairments. He has also founded the Vector Production Unit (UPV) to value the knowledge generated. Currently, the UPV is the first supplier of Ad and AAV vectors to Spanish groups and has the TECNIO Quality accreditation and the ISO9001:2015 accreditation. Finally, in recognition of his career, Dr. M Chillón received the “Transfer Award” in 2018 awarded by the UAB Social Council.
