Hutchinson-Gilford progeria: Splicing-directed therapy in a new mouse model of human accelerated aging

Science Translational Medicine

Volumn 3, Issue 106, 2011, Pages

  Osorio, Fernando G ^a   Navarro, Claire L ^b   Cadiñanos, Juan ^a,h   López Mejía, Isabel C ^c   Quirós, Pedro M ^a   Bartoli, Catherine ^b   Rivera, José ^d   Tazi, Jamal ^c   Guzmán, Gabriela ^e   Varela, Ignacio ^a   Depetris, Danielle ^b   De Carlos, Félix ^f   Cobo, Juan ^f   Andrés, Vicente ^d   De Sandre Giovannoli, Annachiara ^b,g   Freije, José M P ^a   Lévy, Nicolas ^b,g   López Otín, Carlos ^a  

^a DEPARTAMENTO DE BIOQUÍMICA Y BIOLOGÍA MOLECULAR   (Spain)

^b AIX MARSEILLE UNIVERSITY   (France)

^c INSTITUT DE GÉNÉTIQUE MOLÉCULAIRE DE MONTPELLIER   (France)

^d CENTRO NACIONAL DE INVESTIGACIONES CARDIOVASCULARES CNIC   (Spain)

^e HOSPITAL UNIVERSITARIO LA PAZ   (Spain)

^f UNIVERSITY OF OVIEDO   (Spain)

^g TIMONE HOSPITAL   (France)

^h Centro Medico de Asturias   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE MORPHOLINO OLIGONUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE; CELL PROTEIN; HSEX10; HSEX11; MMEX10; MMEX11; PROGERIN; UNCLASSIFIED DRUG; LAMIN A; NUCLEAR PROTEIN; PRELAMIN A; PROTEIN PRECURSOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; BIOACCUMULATION; BONE DISEASE; CARDIOVASCULAR DISEASE; CELL DAMAGE; CELL NUCLEUS; CLINICAL FEATURE; CONTROLLED STUDY; DISEASE MODEL; DRUG EFFICACY; DRUG MECHANISM; EMBRYO; EXON; GENE; GENE MUTATION; HETEROZYGOTE; HISTOPATHOLOGY; HUMAN; HUMAN CELL; LIFESPAN; LMNA GENE; MALE; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PHENOTYPE; PREMATURE AGING; PRIORITY JOURNAL; PROGERIA; RNA SPLICING; TRANSGENIC MOUSE; AGING; ANIMAL; GENETICS; MUTATION; WESTERN BLOTTING;

AGING; ANIMALS; BLOTTING, WESTERN; HUMANS; LAMIN TYPE A; MICE; MUTATION; NUCLEAR PROTEINS; OLIGONUCLEOTIDES, ANTISENSE; PROGERIA; PROTEIN PRECURSORS; RNA SPLICING;

EID: 80054886882     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3002847     Document Type: Article

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45. Acknowledgments: We thank G. Velasco, A. Fueyo, A. Ramsay, J. Valcárcel, M. S. Fernández, P. Roubertoux, M. G. Mattei, C. Tardieu, P. Cau, P. Roll, M. J. Andrés-Manzano, J. M. González, B. Malissen, and L. J. Jiménez-Borreguero for their support and assistance. Funding: This work was supported by grants from Ministerio de Ciencia e Innovación-Spain, PCTI-FICYT Asturias, European Union (FP7 MicroEnviMet), Botín Foundation, Red de Investigación en Enfermedades Cardiovasculares (RECAVA), Institut National de la Santé et de la Recherche Médicale, Agence Nationale de la Recherche, and Association Française contre les Myopathies. The Instituto Universitario de Oncología is supported by Obra Social Cajastur and Acción Transversal del Cáncer-Red Temática de Investigación Cooperativa en Cáncer (RTICC). Author contributions: F.G.O., C.L.N., I.C.L.-M., C.B., A.D.S.-G., and J.T. carried out animal experiments. J. Cadiñanos and J.M.P.F. designed and generated the targeting vector. F.G.O., P.M.Q., I.V., C.L.N., and D.D. performed molecular biology and cell culture-based studies. J.R., G.G., and V.A. carried out cardiovascular analyses. F.d.C. and J. Cobo conducted mCT analysis. C.L.-O., J.M.P.F., and N.L. were responsible for designing and supervising the project. C.L.-O., F.G.O., J.M.P.F., and N.L. wrote the manuscript. Competing interests: The authors declare that they have no competing interests. Accession numbers: RNA expression data are available at the Gene Expression Omnibus repository (accession number GSE32609).