Gene and Cell Therapy

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Gene and Cell Therapy

Dr. Francisco Martín Molina

Information

Scientific interest areas

Gene and Cell Therapy The general aim of this area is the development of new therapeutic strategies for severe diseases by combining adult stem cells and gene transfer Technologies.

Adult stem cells (such as mesenchymal stem cells-MSC) have several properties that make them very attractive for therapy of multiple diseases. In particular their immunomodulatory, secretory and migratory capabilities are of special interest for our Group.

Gene therapy involves the intoduction of genes into cells with therapeutic means. The tools available to deliver the trasngenes are known as gene therapy vectors. We are working with gene therapy vectors derived from HIV-1, one of the most efficient and safe gene delivery vectors for stable expression.

Research areas

    • Design of safer and more efficient gene transfer vectors for Gene Therapy applications. In this direction, we are interested in:
    • 1 – Lentiviral vectors.
      The development of doxycycline-inducible lentiviral vectors based on the original TetR repressor in the absence of transactivators.
      To optimize LVs in order to increase stability and reduce genotoxicity (insulators).
      To develop physiologically regulated LVs.
      To generate human cellular models to study LVs behavior.
    • 2 – Genome edition using specific nucleases (SNs) (zinc finger nucleases (ZFNs) y CRISPR/Cas9). Optimization of SNs delivery into hematopoietic stem cells (HSCs) and T cells.
      Donor DNA designs for improved homologous recombination dependent (HRD) genome edition.
      Alternative system for genome edition independent of HRD.
    • Translation of gene transfer vector technology into clinical applications for rare diseases and cancer. We collaborate with several research institutions, Hospitals and patients association in order to translate our technology to the patients. In particular, we are interested in the development of gene-cell therapy strategies for the treatment of:
    • 1 – Wiskott-Aldrich Syndrome (WAS). We are developing new strategies based on physyologically regulated LVs expressing the correct form of the affected gene (WAS). We are also studying the possibility to implement genome edition as an alternative to lentiviral vectors. We have developed the first WAS pluripotent cellular model for preclinical studies of these therapeutic strategies.
    • 2 – Pompe disease and CoQ deficiency. We aim the development of gene-modified HSCs that over-express the GAA or Coq9 genes as an approach to deliver these proteins into the affected tissues.
    • 3 – Progressive multiple sclerosis. Our strategy consists in the development of mesenchymal stromal cells with improved immunomodulatory and neuroprotective therapeutic activities by expressing different genes.
    • 4 – Limphomas ad Type B leukemias. The strategy is to develop doxycycline inducible CARs-T cells in order to avoid the depletion of B cells during this impressive strategy. We are also interested in modulate CARs expression levels in order to improved efficacy and to reduce secondary effects.

Technology platforms

    • Confocal Microscopy
    • Vector development
    • Cell sorting y FACS analysis
    • Animal facilities
    • Radioactivity
    • Cell Culture
    • Sequencing
    • Molecular cloning
    • Cell biology In vivo imaging
    • Producción vectores Lentivirales
    • Knock-out y knock-in de genes mediante edición genómica (Zinc-Finger Nucleases y Sistema CRISPR/Cas9).

Funding

    • Instituto de Salud Carlos III
    • Consejería de Economía, Innovación y Ciencia de la Junta de Andalucía
    • Consejería de Salud de la Junta de Andalucía.
    • Iniciativa Andaluza de Terapias Avanzadas y la Fundación Progreso y Salud

Research group patents

AUTHORS: Francisco Martin, Pilar Muñoz, Karim Benabdellah, MiguelG. Toscano, Pilar Muñoz y Marién Cobo.

TITLE: HIGHLY INDUCIBLE TET-ON VECTOR SYSTEM

APPLICATION No: PCT / EP2012 / 059408

YEAR: 2011

INSITITUTION: Fundación progreso y Salud (FPS)

COUNTRIES: Europa: EP12727326.6                                             EEUU: US14/118,880

Licenciada a Canvax Biotech en exclusividad

Libertad para comercializar em todo el mundo salvo:

EEUU. Libre de operar  a partir del 26 de junio de 2017

Canadá, Japón, Reino Unido, Francia, Alemania, Lituania y Suiza. Libre de operar  partir de 26/junio/2018

AUTHORS: Francisco Martin, Pilar Muñoz, Karim Benabdellah, MiguelG. Toscano,

TITLE: Vectores lentivirales para la identificación del linaje hematopoyético

APPLICATION No: P201230449 

YEAR: 2012

INSITITUTION: Fundación progreso y Salud (FPS)

COUNTRIES: España. Abandonada en 2015

AUTHORS: Francisco Martin, Karim Benabdellah, Pilar Muñoz, Alejandra Gutierrez Guerrero, Marien Cobo.

TITLE: Insulator to improve gene transfer vectors

APPLICATION No: PCT/EP2014/055027

YEAR: 2014

INSITITUTION: Fundación progreso y Salud (FPS)

COUNTRIES: Europa EP 14716525.2                                             EEUU: US14/776,008

AUTHORS: Per Anderson, Francisco Martin, Ana Carrillo, Marién Cobo.

TITLE: CD105-Negative Multipotent Mesenchymal Stromal Cells

APPLICATION No: EP13382338.5

YEAR: 2014

INSITITUTION: Fundación progreso y Salud (FPS)

COUNTRIES: Europa PCT/EP2014/067996

AUTHORS: Pedro Real Luna, Miguel G. Toscano, Pablo Menendez Bujan, Francisco Martin Molina, Oscar Montero Navarro, Verónica Ayllón Cases, Verónica Ramos Mejía, Clara Bueno Uroz, Marién Cobo Pulido, TamaraRomero Escobar.

TITLE: Método de obtención de megacariocitos y plaquetas

APPLICATION No: P201331568

YEAR: 2014

INSITITUTION: Fundación progreso y Salud (FPS)

COUNTRIES: España

AUTHORS: Per Anderson, Francisco Martin Molina, Ana Belén Carrillo-Gálvez, Marién Cobo Pulido, Angélica García-Perez.

TITLE: Nuevo marcador de superficie de células madre mesenquimales

APPLICATION No: P201331730

YEAR: 2016

INSITITUTION: Fundación progreso y Salud (FPS)

COUNTRIES: España .PCT/ES2014/070874

AUTHORS: Luis Carlos Lopez, Francisco Martin Molina, Eliana Barriocanal Casado, Karim Benabdellah Khlanji, Marien Cobo Puldo, Cristina Cueto Ureña,.

TITLE: Use of Coenzyme Q biosenthetic proteins

APPLICATION No: P201630630

YEAR: 2016

INSITITUTION: Fundación progreso y Salud (FPS) y Universidad de Granada

COUNTRIES: España

Additional information

Collaborations:

Professor Mary Collins . Dean, Faculty of Life Sciences University College London. London (UCL). UK
Professor Francois Loicc-Cosset. Director Human Virology Department. INSERN. ENLS. Lyon. France
Professor Adrian Thrasher. Wellcome Trust Senior Clinical Fellow. Director of Centre for Immunodeficiency. Director of Gene Therapy Programme Institute of Child Health (UCL).
Dr. Michael Holmes. Director of Sangamo Biosciences. Richmond. California.
Prof. Mario Delgado. Profesor de investigación IPB López Neyra. CSIC. Granada
Prof. Ignacio Molina. Vicerrector de investigación PTS. CIBM. Universidad de Granada.
Juan Bueren. Director del Programa de Terapia Génica de Células Hematopoyéticas del CIEMAT

BOOKS AND CHAPTERS

– EDITOR

1-Libro: Gene Therapy-Tools and Potential Applications. Francisco Martin (Ed.)

Ref ISBN: 978-953-51-1014-9,

DOI: 10.5772/50194 FECHA: Febrero 2013

Editorial: InTech, Open Access Available from: http://www.intechopen.com/books/gene-therapy-tools-and-potential-applications

All Chapters download: 63.040

Times cited (Web of Science): 13

– BOOK CHAPTERS

1-Autores: F. Martin, K. Benabdellah, M. Cobo, P. Muñoz, P Anderson MG Toscano

Capitulo: New Vectors for Stable and Safe Gene Modification,

Libro: Gene Therapy – Developments and Future Perspectives. Chunsheng Kang (Ed.)

Ref ISBN: 978-953-307-617-1,

DOI: 10.5772/748 FECHA: Junio 2011

Editorial:InTech, Available from: http://www.intechopen.com/articles/show/title/new-vectors-for-stable-and-safe-gene-modification

Chapter download: 4343

2-Autores: F. Martin, K. A. Gutierrez- Guerrero, K. Benabdellah

Capitulo: Gene Therapy for primary Immunodeficiencies

Libro: Gene Therapy-Tools and Potential Applications. Francisco Martin (Ed.)

Ref ISBN: 978-953-51-1014-9,

DOI: 10.5772/50194 FECHA: Febrero 2013

Editorial:InTech, Open Access Available from: http://www.intechopen.com/books/gene-therapy-tools-and-potential-applications/gene-therapy-for-primary-immunodeficiencies

Chapter download: 1796

3-Autores: F. Martin, K. A. Gutierrez- Guerrero, K. Benabdellah

Capitulo: Gene Delivery Technologies for Efficient Genome Editing: Applications in Gene Therapy

Libro: Modern Tools for Genetic Engineering

Ref ISBN: 978-953-51-2234-0. Print ISBN: 978-953-51-2392-7

DOI: 10.5772/50194 FECHA: May 2016

Editorial:InTech, Open Access Available from: www.intechopen.com/books/modern-tools-for-genetic-engineering

Chapter download: 119

COMMITTEES

Activity: Agencia Nacional de Evaluación y Prospectiva (ANEP)

Entidad: Ministerio de Educación y Ciencia Tema: Evaluador Proyectos

Start date: 2005 End date: —

Activity: Comisión Académica del Programa de doctorado de Biomedicina

Entidad: Universidad de Granada Type of entity: University

Start date: 2012 End date: —

Activity: Comisión de seguimiento de doctorados del Programa de doctorado de Biomedicina

Entidad: Universidad de Granada Type of entity: University

Start date: 2012 End date: —

Activity: Tribunal de evaluación Tesis fin de Master del “Máster de Genética y Evolución”

Entidad: Universidad de Granada Type of entity: University

Start date: 2012 End date: —

Activity: Tribunal de evaluación Tesis fin de Master del “Máster de Inmunología molecular y celular” 

Entidad: Universidad de Granada Type of entity: University

Start date: 2010 End date: —

REVIEWER FOR THE FOLLOWING JOURNALS:

    • Blood
    • Human Molecular Genetic
    • Gene Therapy
    • Human Gene Therapy
    • Stem Cell Research & Therapy
    • Journal of Gene Medicine
    • PlosOne
    • Cell Transplantation
    • BMC Biotechnology
    • Molecular Genetics and Genomics
    • Journal of nanoparticle research
    • Revista de neurología
    • Recent Patents on DNA and Gene Sequence
    • Biotechnology Progress
    • World Journal of Gastroenterology
    • Journal of Pediatric Genetics
    • Clinical and Experimental Immunology.
    • American Journal of Life Science.

REVIEWER FOR THE FOLLOWING ORGANISMS:

    • Swiss National Science Fundation. Suiza. 2002
    • Plan Nacional de Investigación Científica. España (ANEP)
    • Fundaçao para a ciencia e a Tecnologia (FCT). Portugal
    • Agencia para la Calidad del Sistema Universitario de Castilla y León (ACSUCYL)
    • Netherlands Organisation for Health Research and Development. Holanda
    • Comisión de Seguimiento y Control para la Donación y Utilización de Células y Tejidos Humanos
    • LOREAL-UNESCO. Bolsas de investigación 2010

Publications

Selected publications

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Other groups
Area of Genomic Oncology
Area of Genomic Medicine