Institute for Biology and Molecular Genetics
Thomas Schimmang

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Gene and cell therapy

Gene and Cell therapy for Prevention of Hearing Loss

Hearing loss is the most common sensory disorder in humans, its main cause being the loss of cochlear hair cells. We have studied the potential of human mesenchymal stem cells (hMSCs) to differentiate towards hair cells and auditory neurons. hMSCs were first differentiated to neural progenitors and subsequently to hair cell- or auditory neuron-like cells using in vitro culture methods. Differentiation of hMSCs to an intermediate neural progenitor stage was critical for obtaining inner ear sensory lineages. hMSCs generated hair cell-like cells only when neural progenitors derived from nonadherent hMSC cultures grown in serum-free medium were exposed to EGF and retinoic acid. Auditory neuron-like cells were obtained when treated with retinoic acid, and in the presence of defined growth factor combinations containing Sonic Hedgehog.
More recently we have been able to transdifferentiate human fibroblasts towards hair cell-like cells by reprogramming them with transcription factors and corrected a deafness mutation in the human Otoferlin gene using CRIPSR-Cas9. We are now particularly interested to find a direct way for repairing or replacing cells for the human inner ear in vivo.

Recent publications:

  • Generation of inner ear sensory cells from bone marrow-derived human mesenchymal stem cells. Durán Alonso MB, Feijoo-Redondo A, Conde de Felipe M, Carnicero E, García AS, García-Sancho J, Rivolta MN, Giráldez F, Schimmang T.
    Regen Med. 2012 Nov;7(6):769-83. doi: 10.2217/rme.12.65.

  • Cell and tissue therapy in regenerative medicine. Sánchez A, Schimmang T, García-Sancho J.
    Adv Exp Med Biol. 2012;741:89-102. doi: 10.1007/978-1-4614-2098-9_7.

  • Cell- and gene-therapy approaches to inner ear repair. Conde de Felipe MM, Feijoo Redondo A, García-Sancho J, Schimmang T, Durán Alonso MB.
    Histol Histopathol. 2011 Jul;26(7):923-40. doi: 10.14670/HH-26.923. Review.


Instituto de Biología y Genética Molecular
Universidad de Valladolid - Consejo Superior de Investigaciones CIentíficas
C/ Sanz y Forés s/n
47003 - Valladolid - SPAIN
Phone: (+34) 983-184-818 Fax: (+34) 983-184-800

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