sample of publications
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articles
- Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors. Molecular Therapy-Methods & Clinical Development. 27:96-108. 2022
- Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa. MOLECULAR THERAPY. 30:2680-2692. 2022
- FPR2 DNA Aptamers for Targeted Therapy of Wound Repair. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 142:2238-2248. 2022
- Mechanistic interrogation of mutation-independent disease modulators of RDEB identifies the small leucine-rich proteoglycan PRELP as a TGF-beta antagonist and inhibitor of fibrosis. MATRIX BIOLOGY. 111:189-206. 2022
- Non-viral delivery of CRISPR-Cas9 complexes for targeted gene editing via a polymer delivery system. GENE THERAPY. 29:157-170. 2022
- Read-Through for Nonsense Mutations in Type XVII Collagen-Deficient Junctional Epidermolysis Bullosa. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 142:1227-1230.e4. 2022
- Evaluation of systemic gentamicin as translational readthrough therapy for a patient with epidermolysis bullosa simplex with muscular dystrophy owing to PLEC1 pathogenic nonsense variants. JAMA Dermatology. 158:439-443. 2022
- CIBERER: Spanish national network for research on rare diseases: A highly productive collaborative initiative. CLINICAL GENETICS. 101:481-493. 2022
- Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing. MOLECULAR THERAPY. 29:2008-2018. 2021
- Efficient CRISPR-Cas9-mediated gene ablation in human keratinocytes to recapitulate genodermatoses: modeling of Netherton syndrome. Molecular Therapy-Methods & Clinical Development. 18:280-290. 2020
- Advanced statistical techniques for noninvasive hyperglycemic states detection in mice using millimeter-wave spectroscopy. IEEE Transactions on Terahertz Science and Technology. 10:237-245. 2020
- Humanization of tumor stroma by tissue engineering as a tool to improve squamous cell carcinoma xenograft. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. 21:1-10. 2020
- Fibroblast activation and abnormal extracellular matrix remodelling as common hallmarks in three cancer-prone genodermatoses. BRITISH JOURNAL OF DERMATOLOGY. 181:512-522. 2019
- Oxidative stress imbalance as contributing factor in the establishment of fibrosis in recessive dystrophic epidermolysis bullosa. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 139:S631-S631. 2019
- Early, Non-Invasive Sensing of Sustained Hyperglycemia in Mice Using Millimeter-Wave Spectroscopy. SENSORS. 19:1-12. 2019
- Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa. JCI Insight. 4:1-20. 2019
- Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing. MOLECULAR THERAPY. 27:1-13. 2019
- CRISPR/Cas9-Mediated In Situ Correction of LAMB3 Gene in Keratinocytes Derived from a Junctional Epidermolysis Bullosa Patient. MOLECULAR THERAPY. 26:2592-2603. 2018
- Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem Cells. Molecular Therapy-Nucleic Acids. 11:68-78. 2018
- Common mechanisms and signaling pathways in fibroblasts of cancer-prone genodermatoses. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 137:S295-S295. 2017
- Comparative transcriptomic analysis of fibroblasts from two sisters with discordant severe generalized recessive dystrophic epidermolysis bullosa phenotype reveals new molecular markers associated with disease severity. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 137:S212-S212. 2017
- Effects of photodynamic therapy on dermal fibroblasts from xeroderma pigmentosum and Gorlin-Goltz syndrome patients. Oncotarget. 8:77385-77399. 2017
- Classification of skin phenotypes caused by diabetes mellitus using complex scattering parameters in the millimeter-wave frequency range. Scientific Reports. 7:1-8. 2017
- Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 137:836-844. 2017
- In-vivo, non-invasive detection of hyperglycemic states in animal models using mm-wave spectroscopy. Scientific Reports. 6:1-8. 2016
- Long-term skin regeneration in xenografts from iPSC teratoma-derived human keratinocytes. EXPERIMENTAL DERMATOLOGY. 25:736-738. 2016
- Induction of scleroderma fibrosis in skin-humanized mice by anti-Platelet-Derived Growth Factor receptor agonistic autoantibodies. Arthritis & Rheumatology. 68:2263-2273. 2016
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A non-viral gene
therapy for treatment of recessive dystrophic epidermolysis bullosa. EXPERIMENTAL DERMATOLOGY. 25:818-820. 2016 - The transition from linear to highly branched poly(beta-amino ester)s: Branching matters for gene delivery. Science Advances. 2:1-14. 2016
- Differential Features between Chronic Skin Inflammatory Diseases Revealed in Skin-Humanized Psoriasis and Atopic Dermatitis Mouse Models. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 136:136-145. 2016
- Gene editing for the efficient correction of a recurrent COL7A1 mutation in recessive dystrophic epidermolysis bullosa keratinocytes. Molecular Therapy-Nucleic Acids. 5 :1-13. 2016
- Feeder Layer Cell Actions and Applications. Tissue Engineering Part B-Reviews. 21:345-353. 2015
- Innovative therapeutic strategies for recessive dystrophic epidermolysis bullosInnovative therapeutic strategies for recessive dystrophic epidermolysis bullosa. Actas Dermo-Sifiliograficas. 106:376-382. 2015
- Tumor initiation by skin Ha-ras-ment. EXPERIMENTAL DERMATOLOGY. 24:252-253. 2015
- New experimental models of skin homeostasis and diseases. Actas Dermo-Sifiliograficas. 106:17-28. 2015
- Oxidative stress and mitochondrial dysfunction in Kindler syndrome. Orphanet Journal of Rare Diseases. 9:211:1-10. 2014
- Long-term skin regeneration from a gene-targeted human epidermal stem cell clone. MOLECULAR THERAPY. 22:1878-1880. 2014
- Remote diffuse reflectance spectroscopy sensor for tissue engineering monitoring based on blind signal separation. Biomedical Optics Express. 5:3231-3237. 2014
- Tropism-modified AAV vectors overcome barriers to successful cutaneous therapy. MOLECULAR THERAPY. 22:929-939. 2014
- Tissue-engineered oral mucosa for mucosal reconstruction in a pediatric patient with hemifacial microsomia and ankyloglossia. Cleft Palate Craniofacial Journal. 51:146-251. 2014
- Long-term survival of type XVII collagen revertant cells in an animal model of revertant cell therapy. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 134:571-574. 2014
- First Symposium of Ichthyosis Experts = I Jornada de expertos en ictiosis. Actas Dermo-Sifiliograficas. 104:877-882. 2013
- Topical Enzyme-Replacement Therapy Restores Transglutaminase 1 Activity and Corrects Architecture of Transglutaminase-1-Deficient Skin Grafts. AMERICAN JOURNAL OF HUMAN GENETICS. 93:620-630. 2013
- Keratinocyte cell lines derived from severe generalized recessive Epidermolysis Bullosa patients carrying a highly recurrent COL7A1 homozygous mutation: models to assess cell and gene therapies in vitro and in vivo. EXPERIMENTAL DERMATOLOGY. 22:601-603. 2013
- Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. MOLECULAR THERAPY. 21:1695-1704. 2013
- The regenerative potential of fibroblasts in a new diabetes-induced delayed humanised wound healing model. EXPERIMENTAL DERMATOLOGY. 22:195-201. 2013
- A humanized mouse model of HPV-associated pathology driven by E7 expression. PLoS One. 7:1-19. 2012
- Long-Term faithful recapitulation of transglutaminase 1-deficient lamellar ichthyosis in a skin-humanized mouse model, and insights from proteomic studies. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 132:1918-1921. 2012
- Preclinical corrective gene transfer in Xeroderma pigmentosum human skin stem cells. MOLECULAR THERAPY. 20:798-807. 2012
- Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression. HUMAN GENE THERAPY. 23:83-90. 2012
- Risk assessment in skin gene therapy: viral&-cellular fusion transcripts generated by proviral transcriptional read-through in keratinocytes transduced with self-inactivating lentiviral vectors. GENE THERAPY. 18:674-681. 2011
- Development of skin-humanized mouse models of Pachyonychia congenita. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 131:1053-1060. 2011
- Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts. MOLECULAR THERAPY. 19:408-416. 2011
- Development of a bioengineered skin-humanized mouse model for psoriasis: dissecting epidermal-lymphocyte interacting pathways. AMERICAN JOURNAL OF PATHOLOGY. 177:3112-3124. 2010
- In vivo assessment of acute UVB responses in normal and xeroderma pigmentosum (XP-C) skin-humanized mouse models. AMERICAN JOURNAL OF PATHOLOGY. 177:865-872. 2010
- The first COL7A1 mutation survey in a large Spanish Dystrophic Epidermolysis Bullosa cohort: c.6527insC disclosed as an unusually recurrent mutation. BRITISH JOURNAL OF DERMATOLOGY. 163:155-161. 2010
- Human embryonic stem-cell derivatives for full reconstruction of the pluristratified epidermis: a preclinical study. LANCET. 374:1745-1753. 2009
- Correction of laminin-5 deficiency in human epidermal stem cells by transcriptionally targeted lentiviral vectors. MOLECULAR THERAPY. 16:1977-1985. 2008
- Assessment of optimal virus-mediated growth factor gene delivery for human cutaneous wound healing enhancement. JOURNAL OF INVESTIGATIVE DERMATOLOGY. 128:1565-1575. 2008
- In vitro and in vivo wound healing-promoting activities of human cathelicidin LL-37. BRITISH JOURNAL OF DERMATOLOGY. 128:223-236. 2008
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book chapters
- Organotypic and humanized animal models of genodermatoses. In: Skin tissue models. Academic Press. 77-102. 2017
- Current applications for bioengineered skin. In: Translating regenerative medicine to the clinic. Advances in translational medicine. Academic Press. 107-120. 2016
- Bioengineered Skin. In: Skin Biopsy: Perspectives. INTECH. 261-296. 2011
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conference contributions
- Identification of a new molecular modulator of severity in recessive dystrophic epidermolysis bullosa. S211. 2021
- CRISPR/Cas9-based gene editing strategies for clinically-relevant ex vivo correction of Recessive Dystrophic Epidermolysis Bullosa. 59-59. 2020
- Novel players in the establishment and progression of fibrosis in recessive dystrophic epidermolysis bullosa. 71-71. 2020
- Preliminary safety and efficacy outcomes of a phase I trial of systemic mesenchymal stromal cells for recessive dystrophic epidermolysis bullosa. 57-57. 2020
- Development of a Netherton Syndrome model based on targeted deletion of SPINK5 in human keratinocytes. S267-s267. 2019
- Highly efficient gene-editing strategies for clinically-relevant ex vivo correction of Recessive Dystrophic Epidermolysis Bullosa in primary patient cells 2019
- Preclinical models for in vivo gene editing of COL7A1 based on delivery of CRISPR/Cas9 to RDEB patient skin by adenoviral vectors 2019
- Clinically-relevant correction of recessive dystrophic epidermolysis bullosa by dual sgRNA CRISPR/Cas9-mediated gene editing. A116-A117. 2018
- Disease Severity Modulators in Recessive Dystrophic Epidermolysis Bullosa. 31-32. 2018
- Non-invasive Detection and Monitoring of Sustained Glycemic Fluctuations using Mm-Wave Spectroscopy 2018
- Determination of hyperglycemic states in mice using millimeter-wave sensing 2017
- Gene editing-mediated excision of mutation-bearing exon 80 of COL7A1 gene for the efficient correction of recessive dystrophic epidermolysis bullosa patient derived-epidermalstem cells 2017
- Monitoring the evolution of hyperglycemia in mice using mm-wave spectroscopy 2017
- Fluorescence Spectroscopic Instrument for the Identification of Hyperglycemia 2016
- Fluorescence spectroscopic instrument for the identification of hyperglycemia. OTu2C.6. 2016
- Novedades en el tratamiento de la Epidermolisis Bullosa distrófica /Advances in DEB treatment 2016
- Síndrome de Netherton: de los modelos animales, al paciente. Ensayo clínico de terapia génica, para Síndrome de Netherton 2016
- Bioresorbable nanoparticles for drug delivery and targeting. Preparation and application in wound healing. 29-29. 2015
- Targeted Gene Therapy Approaches for RDEB Due to a Highly Recurrent COL7A1 "Spanish" Mutation 2015
- Assessment of the performance and fate of adipose-derived mesenchymal stem cells forming part of bioengineered skin equivalents in vitro and in vivo. 99. 2014
- Identificación de nuevos mecanismos fisiopatológicos en el síndrome de Kindler 2014
- Cell-based therapy for diabetic wounds: from the bench to the patient 2013
- Development of bioengineered skin-humanized mouse models for inflammatory skin diseases. 129-129. 2013
- Fate and function of adipose-derived mesenchymal stem cells (ADMSC) in bioengineered skin equivalents in vitro and in vivo. 129-129. 2013
- Long-term COLVII expression after histocompatible bioengineered skin transplantation in a COL VII-null RDEB patient 2013
- Role of mitochondria in the pathogenesis of kindler syndrome. 130-130. 2013
- Cutaneous gene therapy approach for the correction of congenital generalized lipodystrophy. 98-98. 2012
- Establishment and characterization of keratinocyte cell lines derived from RDEB patients to assess cell and gene therapies in vitro and in vivo. 98-98. 2012
- Potential role of oxidative stress from mitochondria in Kindler Syndrome. 82-82. 2012
- Topical enzyme replacement therapy restores transglutaminase 1 activity and corrects architecture of transglutaminase 1 deficient skin grafts. 89-89. 2012
- Development of a skin humanized mouse model for transglutaminase 1 deficient lamellar ichthyosis. 71-71. 2011
- Generación de piel a partir de células madre epidérmicas: estudios preclínicos y extrapolaciones clínicas 2011
- Human fibroblasts in skin regeneration: A preclinical study in diabetic wound healing 2011
- Human fibroblasts in skin regeneration: A preclinical study in diabetic wound healing. 2-2. 2011
- In vitro disadvantage of type XVII collagen revertant keratinocytes when cultured for revertant cell therapy. 69-69. 2011
- In vivo assessment of low-and high-dose of UVB irradiation on xeroderma pigmentosum (XP-C) and differentially pigmented human bioengineered skin. 65-65. 2011
- In vivo efficacy of human mesenchymal stem cell transplantation in bioingeneered skin-humanized mouse model for psoriasis. 9-9. 2011
- In vivo efficacy of human mesenchymal stem cell transplantation in bioingeneered skin-humanized mouse model for psoriasis 2011