Gene therapy for primary immunodeficiencies: Part 2 (Articolo in rivista)

Type

• Prodotto della ricerca (Classe)
• Articolo in rivista (Classe)

Label

• Gene therapy for primary immunodeficiencies: Part 2 (Articolo in rivista) (literal)

Anno

• 2012-01-01T00:00:00+01:00 (literal)

Alternative label

• Alessandro Aiuti1, 2, Rosa Bacchetta1, Reinhard Seger3, Anna Villa1, 4, Marina Cavazzana-Calvo5, 6, 7, 8 (2012)
  Gene therapy for primary immunodeficiencies: Part 2
  in Current opinion in immunology
  (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#autori

• Alessandro Aiuti1, 2, Rosa Bacchetta1, Reinhard Seger3, Anna Villa1, 4, Marina Cavazzana-Calvo5, 6, 7, 8 (literal)

Pagina inizio

• 585 (literal)

Pagina fine

• 591 (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroVolume

• 24 (literal)

Rivista

• Current opinion in immunology (Rivista)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroFascicolo

• 5 (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#affiliazioni

• 1 San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), Division of Regenerative Medicine, San Raffaele Scientific Institute, Milan, Italy 2 Department of Pediatrics, University of Rome Tor Vergata, Children's Hospital Bambino Gesù, Rome, Italy 3 Division of Immunology/Haematology/BMT, University Children's Hospital of Zurich, Zurich, Switzerland 4 Institute of Genetic and Biomedical Research, Milan Unit, National Research Council (CNR), Italy 5 U768 INSERM, Paris, France 6 Département de Biothérapie, AP-HP, Hopital Universitaire Necker - Enfants Malades, Paris, France 7 Université Paris Descartes, Sorbonne Paris Cité, IMAGINE Institute, Paris, France 8 CIC Biothérapie GHU Ouest, INSERM-APHP, Paris, France (literal)

Titolo

• Gene therapy for primary immunodeficiencies: Part 2 (literal)

Abstract

• Gene therapy has become an attractive alternative therapeutic strategy to allogeneic transplant for primary immunodeficiencies (PIDs) owing to known genetic defects. Clinical trials using gammaretroviral vectors have demonstrated the proof of principle of gene therapy for Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD), but have also highlighted limitations of the technology. New strategies based on vectors that can achieve more robust correction with less risk of insertional mutagenesis are being developed. In this review we present the status of gene therapy for WAS and CGD, and discuss the emerging application of similar strategies to a broader range of PIDs, such as IPEX syndrome. (literal)

Prodotto di

• Cellule staminali pluripotenti indotte per la terapia di malattie genetiche (ME.P05.020.001) (Modulo)
• Istituto di Ricerca Genetica e Biomedica (IRGB) (Istituto)

Autore CNR

• ANNA VILLA (Unità di personale interno)

Incoming links:

Autore CNR di

• ANNA VILLA (Unità di personale interno)

Prodotto

• Istituto di Ricerca Genetica e Biomedica (IRGB) (Istituto)
• Cellule staminali pluripotenti indotte per la terapia di malattie genetiche (ME.P05.020.001) (Modulo)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#rivistaDi

• Current opinion in immunology (Rivista)