• 121 Citations
  • 5 h-Index
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Fingerprint Dive into the research topics where Stefania Giannelli is active. These topic labels come from the works of this person. Together they form a unique fingerprint.

  • 12 Similar Profiles
Genetic Therapy Medicine & Life Sciences
Hematopoietic Stem Cells Medicine & Life Sciences
Gene therapy Chemical Compounds
Lymphopenia Medicine & Life Sciences
Wiskott-Aldrich Syndrome Medicine & Life Sciences
Stem cells Chemical Compounds
Transplants Chemical Compounds
Cell- and Tissue-Based Therapy Medicine & Life Sciences

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Research Output 2015 2019

  • 121 Citations
  • 5 h-Index
  • 10 Article
11 Citations (Scopus)

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study

Ferrua, F., Cicalese, M. P., Galimberti, S., Giannelli, S., Dionisio, F., Barzaghi, F., Migliavacca, M., Bernardo, M. E., Calbi, V., Assanelli, A. A., Facchini, M., Fossati, C., Albertazzi, E., Scaramuzza, S., Brigida, I., Scala, S., Basso-Ricci, L., Pajno, R., Casiraghi, M., Canarutto, D. & 18 others, Salerio, F. A., Albert, M. H., Bartoli, A., Wolf, H. M., Fiori, R., Silvani, P., Gattillo, S., Villa, A., Biasco, L., Dott, C., Culme-Seymour, E. J., van Rossem, K., Atkinson, G., Valsecchi, M. G., Roncarolo, M. G., Ciceri, F., Naldini, L. & Aiuti, A., May 1 2019, In : The Lancet Haematology. 6, 5, p. e239-e253

Research output: Contribution to journalArticle

Open Access
Wiskott-Aldrich Syndrome
Cell- and Tissue-Based Therapy
Genetic Therapy
Stem Cells
Wiskott-Aldrich Syndrome Protein

Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery

Giannelli, SG., Luoni, M., Castoldi, V., Massimino, L., Cabassi, T., Angeloni, D., Demontis, GC., Leocani, L., Andreazzoli, M. & Broccoli, V., 2018, In : Human Molecular Genetics. 27, 5, p. 761-779 19 p.

Research output: Contribution to journalArticle

Retinitis Pigmentosa
Clustered Regularly Interspaced Short Palindromic Repeats

Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans

Scala, S., Basso-Ricci, L., Dionisio, F., Pellin, D., Giannelli, S., Salerio, FA., Leonardelli, L., Cicalese, MP., Ferrua, F., Aiuti, A. & Biasco, L., 2018, In : Nature Medicine. 24, 11, p. 1683-1690 8 p.

Research output: Contribution to journalArticle

Autologous Transplantation
Hematopoietic Stem Cells
Stem cells
Gene therapy

Gene therapy in rare diseases: The benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID

Stirnadel-Farrant, H., Kudari, M., Garman, N., Imrie, J., Chopra, B., Giannelli, S., Gabaldo, M., Corti, A., Zancan, S., Aiuti, A., Cicalese, MP., Batta, R., Appleby, J., Davinelli, M. & Ng, P., 2018, In : Orphanet Journal of Rare Diseases. 13, 1, 49.

Research output: Contribution to journalArticle

Adenosine Deaminase
Rare Diseases
Genetic Therapy

T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency

Brigida, I., Zoccolillo, M., Cicalese, MP., Pfajfer, L., Barzaghi, F., Scala, S., Oleaga-Quintas, C., Álvarez-Álvarez, JA., Sereni, L., Giannelli, S., Sartirana, C., Dionisio, F., Pavesi, L., Benavides-Nieto, M., Basso-Ricci, L., Capasso, P., Mazzi, B., Rosain, J., Marcus, N., Lee, YN. & 26 others, Somech, R., Degano, M., Raiola, G., Caorsi, R., Picco, P., Velez, MM., Khourieh, J., Arias, AA., Bousfiha, A., Issekutz, T., Issekutz, A., Boisson, B., Dobbs, K., Villa, A., Lombardo, A., Neven, B., Moshous, D., Casanova, JL., Franco, JL., Notarangelo, LD., Scielzo, C., Volpi, S., Dupré, L., Bustamante, J., Gattorno, M. & Aiuti, A., 2018, In : Blood. 132, 22, p. 2362-2374 13 p.

Research output: Contribution to journalArticle

Germ-Line Mutation
T-Cell Antigen Receptor