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Items: 1 to 50 of 171

1.

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.

Nat Med. 2018 Oct 1. doi: 10.1038/s41591-018-0195-3. [Epub ahead of print]

PMID:
30275570
2.

T cell defects in patients with ARPC1B germline mutations account for their 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, Somech R, Degano M, Raiola G, Caorsi R, Picco P, Moncada Velez M, 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.

Blood. 2018 Sep 25. pii: blood-2018-07-863431. doi: 10.1182/blood-2018-07-863431. [Epub ahead of print]

PMID:
30254128
3.

Lymphocytes are a major source of circulating soluble dipeptidyl peptidase 4.

Casrouge A, Sauer AV, Barreira da Silva R, Tejera-Alhambra M, Sánchez-Ramón S, ICAReB, Cancrini C, Ingersoll MA, Aiuti A, Albert ML.

Clin Exp Immunol. 2018 Sep 24. doi: 10.1111/cei.13163. [Epub ahead of print] Review.

PMID:
30251416
4.

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.

Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, Gaspar HB, Notarangelo LD, Grunebaum E.

J Allergy Clin Immunol. 2018 Sep 5. pii: S0091-6749(18)31268-5. doi: 10.1016/j.jaci.2018.08.024. [Epub ahead of print]

PMID:
30194989
5.

Circulating Follicular Helper and Follicular Regulatory T Cells Are Severely Compromised in Human CD40 Deficiency: A Case Report.

Cicalese MP, Gerosa J, Baronio M, Montin D, Licciardi F, Soresina A, Dellepiane RM, Miano M, Baselli LA, Volpi S, Dufour C, Plebani A, Aiuti A, Lougaris V, Fousteri G.

Front Immunol. 2018 Aug 6;9:1761. doi: 10.3389/fimmu.2018.01761. eCollection 2018.

6.

Next-Generation Sequencing Reveals A JAGN1 Mutation in a Syndromic Child With Intermittent Neutropenia.

Cifaldi C, Serafinelli J, Petricone D, Brigida I, Di Cesare S, Di Matteo G, Chiriaco M, De Vito R, Palumbo G, Rossi P, Palma P, Cancrini C, Aiuti A, Finocchi A.

J Pediatr Hematol Oncol. 2018 Jul 23. doi: 10.1097/MPH.0000000000001256. [Epub ahead of print]

PMID:
30044346
7.

JAK3 mutations in Italian patients affected by SCID: New molecular aspects of a long-known gene.

Di Matteo G, Chiriaco M, Scarselli A, Cifaldi C, Livadiotti S, Di Cesare S, Ferradini V, Aiuti A, Rossi P, Finocchi A, Cancrini C.

Mol Genet Genomic Med. 2018 Sep;6(5):713-721. doi: 10.1002/mgg3.391. Epub 2018 Jul 21.

8.

Successful treatment with Harvoni® in an ADA-SCID infant with HCV infection allowed gene therapy with Strimvelis®.

Tucci F, Calbi V, Barzaghi F, Migliavacca M, Ferrua F, Bernardo ME, Canarutto D, Consiglieri G, Recupero S, Calzatini F, Gabaldo M, Lucano C, Casiraghi M, Darin S, Dionisio F, Marktel S, Cestone E, Finazzi R, Mieli-Vergani G, Boeri E, Appleby J, Elaziz DA, Ciceri F, Aiuti A, Cicalese MP.

Hepatology. 2018 Jul 16. doi: 10.1002/hep.30160. [Epub ahead of print]

PMID:
30014500
9.

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.

Orphanet J Rare Dis. 2018 Apr 6;13(1):49. doi: 10.1186/s13023-018-0791-9.

10.

Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry.

Maccari ME, Abolhassani H, Aghamohammadi A, Aiuti A, Aleinikova O, Bangs C, Baris S, Barzaghi F, Baxendale H, Buckland M, Burns SO, Cancrini C, Cant A, Cathébras P, Cavazzana M, Chandra A, Conti F, Coulter T, Devlin LA, Edgar JDM, Faust S, Fischer A, Garcia-Prat M, Hammarström L, Heeg M, Jolles S, Karakoc-Aydiner E, Kindle G, Kiykim A, Kumararatne D, Grimbacher B, Longhurst H, Mahlaoui N, Milota T, Moreira F, Moshous D, Mukhina A, Neth O, Neven B, Nieters A, Olbrich P, Ozen A, Pachlopnik Schmid J, Picard C, Prader S, Rae W, Reichenbach J, Rusch S, Savic S, Scarselli A, Scheible R, Sediva A, Sharapova SO, Shcherbina A, Slatter M, Soler-Palacin P, Stanislas A, Suarez F, Tucci F, Uhlmann A, van Montfrans J, Warnatz K, Williams AP, Wood P, Kracker S, Condliffe AM, Ehl S.

Front Immunol. 2018 Mar 16;9:543. doi: 10.3389/fimmu.2018.00543. eCollection 2018.

11.

First Occurrence of Plasmablastic Lymphoma in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Disease Patient and Review of the Literature.

Migliavacca M, Assanelli A, Ponzoni M, Pajno R, Barzaghi F, Giglio F, Ferrua F, Frittoli M, Brigida I, Dionisio F, Nicoletti R, Casiraghi M, Roncarolo MG, Doglioni C, Peccatori J, Ciceri F, Cicalese MP, Aiuti A.

Front Immunol. 2018 Feb 2;9:113. doi: 10.3389/fimmu.2018.00113. eCollection 2018.

12.

Neutrophils drive type I interferon production and autoantibodies in patients with Wiskott-Aldrich syndrome.

Cervantes-Luevano KE, Caronni N, Castiello MC, Fontana E, Piperno GM, Naseem A, Uva P, Bosticardo M, Marcovecchio GE, Notarangelo LD, Cicalese MP, Aiuti A, Villa A, Benvenuti F.

J Allergy Clin Immunol. 2018 Feb 13. pii: S0091-6749(18)30207-0. doi: 10.1016/j.jaci.2017.11.063. [Epub ahead of print]

PMID:
29447842
13.

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.

Cicalese MP, Ferrua F, Castagnaro L, Rolfe K, De Boever E, Reinhardt RR, Appleby J, Roncarolo MG, Aiuti A.

Mol Ther. 2018 Mar 7;26(3):917-931. doi: 10.1016/j.ymthe.2017.12.022. Epub 2018 Jan 4.

14.

Autonomous role of Wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation.

Sereni L, Castiello MC, Marangoni F, Anselmo A, di Silvestre D, Motta S, Draghici E, Mantero S, Thrasher AJ, Giliani S, Aiuti A, Mauri P, Notarangelo LD, Bosticardo M, Villa A.

J Allergy Clin Immunol. 2018 Oct;142(4):1272-1284. doi: 10.1016/j.jaci.2017.12.1000. Epub 2018 Feb 6.

PMID:
29421274
15.

Impaired X-CGD T cell compartment is gp91phox-NADPH oxidase independent.

Chiriaco M, Casciano F, Di Matteo G, Gentner B, Claps A, Di Cesare S, Cotugno N, D'Argenio P, Rossi P, Aiuti A, Finocchi A.

Clin Immunol. 2018 Aug;193:52-59. doi: 10.1016/j.clim.2018.01.010. Epub 2018 Feb 3.

PMID:
29410324
16.

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.

Calbi V, Fumagalli F, Consiglieri G, Penati R, Acquati S, Redaelli D, Attanasio V, Marcella F, Cicalese MP, Migliavacca M, Barzaghi F, Ferrua F, Assanelli A, Silvani P, Zoccolillo M, Chidini G, Chiesa R, Arora R, Ciotti F, Sarzana M, Antonioli G, Baldoli C, Morena F, Martino S, Ardissino GL, Sora MGN, Naldini L, Ciceri F, Aiuti A, Bernardo ME.

Bone Marrow Transplant. 2018 Jul;53(7):913-917. doi: 10.1038/s41409-017-0085-1. Epub 2018 Jan 29. No abstract available.

17.

Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

Barzaghi F, Amaya Hernandez LC, Neven B, Ricci S, Kucuk ZY, Bleesing JJ, Nademi Z, Slatter MA, Ulloa ER, Shcherbina A, Roppelt A, Worth A, Silva J, Aiuti A, Murguia-Favela L, Speckmann C, Carneiro-Sampaio M, Fernandes JF, Baris S, Ozen A, Karakoc-Aydiner E, Kiykim A, Schulz A, Steinmann S, Notarangelo LD, Gambineri E, Lionetti P, Shearer WT, Forbes LR, Martinez C, Moshous D, Blanche S, Fisher A, Ruemmele FM, Tissandier C, Ouachee-Chardin M, Rieux-Laucat F, Cavazzana M, Qasim W, Lucarelli B, Albert MH, Kobayashi I, Alonso L, Diaz De Heredia C, Kanegane H, Lawitschka A, Seo JJ, Gonzalez-Vicent M, Diaz MA, Goyal RK, Sauer MG, Yesilipek A, Kim M, Yilmaz-Demirdag Y, Bhatia M, Khlevner J, Richmond Padilla EJ, Martino S, Montin D, Neth O, Molinos-Quintana A, Valverde-Fernandez J, Broides A, Pinsk V, Ballauf A, Haerynck F, Bordon V, Dhooge C, Garcia-Lloret ML, Bredius RG, Kałwak K, Haddad E, Seidel MG, Duckers G, Pai SY, Dvorak CC, Ehl S, Locatelli F, Goldman F, Gennery AR, Cowan MJ, Roncarolo MG, Bacchetta R; Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT).

J Allergy Clin Immunol. 2018 Mar;141(3):1036-1049.e5. doi: 10.1016/j.jaci.2017.10.041. Epub 2017 Dec 11.

18.

Severe Toxoplasma gondii infection in a member of a NFKB2-deficient family with T and B cell dysfunction.

Maccari ME, Scarselli A, Di Cesare S, Floris M, Angius A, Deodati A, Chiriaco M, Cambiaso P, Corrente S, Colafati GS, Utz PJ, Angelini F, Fierabracci A, Aiuti A, Carsetti R, Rosenberg JM, Cappa M, Rossi P, Bacchetta R, Cancrini C.

Clin Immunol. 2017 Oct;183:273-277. doi: 10.1016/j.clim.2017.09.011. Epub 2017 Sep 14. No abstract available.

PMID:
28919517
19.

Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug.

Ferrua F, Aiuti A.

Hum Gene Ther. 2017 Nov;28(11):972-981. doi: 10.1089/hum.2017.175. Review.

PMID:
28847159
20.

A map of human circular RNAs in clinically relevant tissues.

Maass PG, Glažar P, Memczak S, Dittmar G, Hollfinger I, Schreyer L, Sauer AV, Toka O, Aiuti A, Luft FC, Rajewsky N.

J Mol Med (Berl). 2017 Nov;95(11):1179-1189. doi: 10.1007/s00109-017-1582-9. Epub 2017 Aug 25.

21.

Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I.

Azario I, Pievani A, Del Priore F, Antolini L, Santi L, Corsi A, Cardinale L, Sawamoto K, Kubaski F, Gentner B, Bernardo ME, Valsecchi MG, Riminucci M, Tomatsu S, Aiuti A, Biondi A, Serafini M.

Sci Rep. 2017 Aug 25;7(1):9473. doi: 10.1038/s41598-017-09958-9.

22.

Biological and functional characterization of bone marrow-derived mesenchymal stromal cells from patients affected by primary immunodeficiency.

Starc N, Ingo D, Conforti A, Rossella V, Tomao L, Pitisci A, De Mattia F, Brigida I, Algeri M, Montanari M, Palumbo G, Merli P, Rossi P, Aiuti A, Locatelli F, Bernardo ME.

Sci Rep. 2017 Aug 15;7(1):8153. doi: 10.1038/s41598-017-08550-5.

23.

Long-Term Outcome of Adenosine Deaminase-Deficient Patients-a Single-Center Experience.

Scott O, Kim VH, Reid B, Pham-Huy A, Atkinson AR, Aiuti A, Grunebaum E.

J Clin Immunol. 2017 Aug;37(6):582-591. doi: 10.1007/s10875-017-0421-7. Epub 2017 Jul 26.

PMID:
28748310
24.

Multiparametric Whole Blood Dissection: A one-shot comprehensive picture of the human hematopoietic system.

Basso-Ricci L, Scala S, Milani R, Migliavacca M, Rovelli A, Bernardo ME, Ciceri F, Aiuti A, Biasco L.

Cytometry A. 2017 Oct;91(10):952-965. doi: 10.1002/cyto.a.23148. Epub 2017 Jun 13.

25.

Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.

Penati R, Fumagalli F, Calbi V, Bernardo ME, Aiuti A.

J Inherit Metab Dis. 2017 Jul;40(4):543-554. doi: 10.1007/s10545-017-0052-4. Epub 2017 May 30. Review.

26.

Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products.

Aiuti A, Roncarolo MG, Naldini L.

EMBO Mol Med. 2017 Jun;9(6):737-740. doi: 10.15252/emmm.201707573. No abstract available.

27.

Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy.

Zonari E, Desantis G, Petrillo C, Boccalatte FE, Lidonnici MR, Kajaste-Rudnitski A, Aiuti A, Ferrari G, Naldini L, Gentner B.

Stem Cell Reports. 2017 Apr 11;8(4):977-990. doi: 10.1016/j.stemcr.2017.02.010. Epub 2017 Mar 16.

28.

Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice.

Carriglio N, Klapwijk J, Hernandez RJ, Vezzoli M, Chanut F, Lowe R, Draghici E, Nord M, Albertini P, Cristofori P, Richards J, Staton H, Appleby J, Aiuti A, Sauer AV.

Hum Gene Ther Clin Dev. 2017 Mar;28(1):17-27. doi: 10.1089/humc.2016.191. Erratum in: Hum Gene Ther Clin Dev. 2017 Jun;28(2):116.

PMID:
28319446
29.

Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients.

Sauer AV, Hernandez RJ, Fumagalli F, Bianchi V, Poliani PL, Dallatomasina C, Riboni E, Politi LS, Tabucchi A, Carlucci F, Casiraghi M, Carriglio N, Cominelli M, Forcellini CA, Barzaghi F, Ferrua F, Minicucci F, Medaglini S, Leocani L, la Marca G, Notarangelo LD, Azzari C, Comi G, Baldoli C, Canale S, Sessa M, D'Adamo P, Aiuti A.

Sci Rep. 2017 Jan 11;7:40136. doi: 10.1038/srep40136.

30.

Bone marrow-derived CD34- fraction: A rich source of mesenchymal stromal cells for clinical application.

Ingo DM, Redaelli D, Rossella V, Perini O, Santoleri L, Ciceri F, Aiuti A, Bernardo ME.

Cytotherapy. 2016 Dec;18(12):1560-1563. doi: 10.1016/j.jcyt.2016.08.011. Epub 2016 Oct 11. No abstract available.

PMID:
27742233
31.

AQP8 transports NOX2-generated H2O2 across the plasma membrane to promote signaling in B cells.

Bertolotti M, Farinelli G, Galli M, Aiuti A, Sitia R.

J Leukoc Biol. 2016 Nov;100(5):1071-1079. Epub 2016 Jun 2.

PMID:
27256569
32.

A prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysis.

Speckmann C, Doerken S, Aiuti A, Albert MH, Al-Herz W, Allende LM, Scarselli A, Avcin T, Perez-Becker R, Cancrini C, Cant A, Di Cesare S, Finocchi A, Fischer A, Gaspar HB, Ghosh S, Gennery A, Gilmour K, González-Granado LI, Martinez-Gallo M, Hambleton S, Hauck F, Hoenig M, Moshous D, Neven B, Niehues T, Notarangelo L, Picard C, Rieber N, Schulz A, Schwarz K, Seidel MG, Soler-Palacin P, Stepensky P, Strahm B, Vraetz T, Warnatz K, Winterhalter C, Worth A, Fuchs S, Uhlmann A, Ehl S; P-CID study of the Inborn Errors Working Party of the EBMT.

J Allergy Clin Immunol. 2017 Apr;139(4):1302-1310.e4. doi: 10.1016/j.jaci.2016.07.040. Epub 2016 Sep 19.

PMID:
27658761
33.

Large Deletion of MAGT1 Gene in a Patient with Classic Kaposi Sarcoma, CD4 Lymphopenia, and EBV Infection.

Brigida I, Chiriaco M, Di Cesare S, Cittaro D, Di Matteo G, Giannelli S, Lazarevic D, Zoccolillo M, Stupka E, Jenkner A, Francalanci P, Livadiotti S, Morawski A, Ravell J, Lenardo M, Cancrini C, Aiuti A, Finocchi A.

J Clin Immunol. 2017 Jan;37(1):32-35. doi: 10.1007/s10875-016-0341-y. Epub 2016 Oct 21. No abstract available.

34.

Primary immunodeficiency diseases: Genomic approaches delineate heterogeneous Mendelian disorders.

Stray-Pedersen A, Sorte HS, Samarakoon P, Gambin T, Chinn IK, Coban Akdemir ZH, Erichsen HC, Forbes LR, Gu S, Yuan B, Jhangiani SN, Muzny DM, Rødningen OK, Sheng Y, Nicholas SK, Noroski LM, Seeborg FO, Davis CM, Canter DL, Mace EM, Vece TJ, Allen CE, Abhyankar HA, Boone PM, Beck CR, Wiszniewski W, Fevang B, Aukrust P, Tjønnfjord GE, Gedde-Dahl T, Hjorth-Hansen H, Dybedal I, Nordøy I, Jørgensen SF, Abrahamsen TG, Øverland T, Bechensteen AG, Skogen V, Osnes LTN, Kulseth MA, Prescott TE, Rustad CF, Heimdal KR, Belmont JW, Rider NL, Chinen J, Cao TN, Smith EA, Caldirola MS, Bezrodnik L, Lugo Reyes SO, Espinosa Rosales FJ, Guerrero-Cursaru ND, Pedroza LA, Poli CM, Franco JL, Trujillo Vargas CM, Aldave Becerra JC, Wright N, Issekutz TB, Issekutz AC, Abbott J, Caldwell JW, Bayer DK, Chan AY, Aiuti A, Cancrini C, Holmberg E, West C, Burstedt M, Karaca E, Yesil G, Artac H, Bayram Y, Atik MM, Eldomery MK, Ehlayel MS, Jolles S, Flatø B, Bertuch AA, Hanson IC, Zhang VW, Wong LJ, Hu J, Walkiewicz M, Yang Y, Eng CM, Boerwinkle E, Gibbs RA, Shearer WT, Lyle R, Orange JS, Lupski JR.

J Allergy Clin Immunol. 2017 Jan;139(1):232-245. doi: 10.1016/j.jaci.2016.05.042. Epub 2016 Jul 16. Erratum in: J Allergy Clin Immunol. 2018 Feb;141(2):832.

35.

The Role of Conditioning in Hematopoietic Stem-Cell Gene Therapy.

Bernardo ME, Aiuti A.

Hum Gene Ther. 2016 Oct;27(10):741-748. doi: 10.1089/hum.2016.103. Review.

PMID:
27530055
36.

Lentiviral Vector Gene Therapy Protects XCGD Mice From Acute Staphylococcus aureus Pneumonia and Inflammatory Response.

Farinelli G, Jofra Hernandez R, Rossi A, Ranucci S, Sanvito F, Migliavacca M, Brombin C, Pramov A, Di Serio C, Bovolenta C, Gentner B, Bragonzi A, Aiuti A.

Mol Ther. 2016 Oct;24(10):1873-1880. doi: 10.1038/mt.2016.150. Epub 2016 Jul 26.

37.

Safer conditioning for blood stem cell transplants.

Aiuti A, Naldini L.

Nat Biotechnol. 2016 Jul 12;34(7):721-3. doi: 10.1038/nbt.3629. No abstract available.

PMID:
27404882
38.

Immunotherapy of acute leukemia by chimeric antigen receptor-modified lymphocytes using an improved Sleeping Beauty transposon platform.

Magnani CF, Turazzi N, Benedicenti F, Calabria A, Tenderini E, Tettamanti S, Giordano Attianese GM, Cooper LJ, Aiuti A, Montini E, Biondi A, Biagi E.

Oncotarget. 2016 Aug 9;7(32):51581-51597. doi: 10.18632/oncotarget.9955.

39.

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.

Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, Fiori R, Silvani P, Rancoita PM, Gabaldo M, Benedicenti F, Antonioli G, Assanelli A, Cicalese MP, Del Carro U, Sora MG, Martino S, Quattrini A, Montini E, Di Serio C, Ciceri F, Roncarolo MG, Aiuti A, Naldini L, Biffi A.

Lancet. 2016 Jul 30;388(10043):476-87. doi: 10.1016/S0140-6736(16)30374-9. Epub 2016 Jun 8.

PMID:
27289174
40.

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases.

Biasco L, Pellin D, Scala S, Dionisio F, Basso-Ricci L, Leonardelli L, Scaramuzza S, Baricordi C, Ferrua F, Cicalese MP, Giannelli S, Neduva V, Dow DJ, Schmidt M, Von Kalle C, Roncarolo MG, Ciceri F, Vicard P, Wit E, Di Serio C, Naldini L, Aiuti A.

Cell Stem Cell. 2016 Jul 7;19(1):107-19. doi: 10.1016/j.stem.2016.04.016. Epub 2016 May 26.

41.

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Cicalese MP, Ferrua F, Castagnaro L, Pajno R, Barzaghi F, Giannelli S, Dionisio F, Brigida I, Bonopane M, Casiraghi M, Tabucchi A, Carlucci F, Grunebaum E, Adeli M, Bredius RG, Puck JM, Stepensky P, Tezcan I, Rolfe K, De Boever E, Reinhardt RR, Appleby J, Ciceri F, Roncarolo MG, Aiuti A.

Blood. 2016 Jul 7;128(1):45-54. doi: 10.1182/blood-2016-01-688226. Epub 2016 Apr 29. Erratum in: Blood. 2017 Jun 15;129(24):3271.

42.

A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation.

Brigida I, Scaramuzza S, Lazarevic D, Cittaro D, Ferrua F, Leonardelli L, Alessio M, Forma O, Lanzani C, Viarengo G, Ciceri F, Jankovic M, Pesce F, Aiuti A, Cicalese MP.

J Allergy Clin Immunol. 2016 Aug;138(2):619-622.e7. doi: 10.1016/j.jaci.2016.03.007. Epub 2016 Apr 22. No abstract available.

43.

Pioglitazone as a novel therapeutic approach in chronic granulomatous disease.

Migliavacca M, Assanelli A, Ferrua F, Cicalese MP, Biffi A, Frittoli M, Silvani P, Chidini G, Calderini E, Mandelli A, Camporesi A, Milani R, Farinelli G, Nicoletti R, Ciceri F, Aiuti A, Bernardo ME.

J Allergy Clin Immunol. 2016 Jun;137(6):1913-1915.e2. doi: 10.1016/j.jaci.2016.01.033. Epub 2016 Apr 4. No abstract available.

44.

The case of an APDS patient: Defects in maturation and function and decreased in vitro anti-mycobacterial activity in the myeloid compartment.

Chiriaco M, Brigida I, Ariganello P, Di Cesare S, Di Matteo G, Taus F, Cittaro D, Lazarevic D, Scarselli A, Santilli V, Attardi E, Stupka E, Giannelli S, Fraziano M, Finocchi A, Rossi P, Aiuti A, Palma P, Cancrini C.

Clin Immunol. 2017 May;178:20-28. doi: 10.1016/j.clim.2015.12.008. Epub 2015 Dec 28.

PMID:
26732860
45.

Tracking genetically engineered lymphocytes long-term reveals the dynamics of T cell immunological memory.

Oliveira G, Ruggiero E, Stanghellini MT, Cieri N, D'Agostino M, Fronza R, Lulay C, Dionisio F, Mastaglio S, Greco R, Peccatori J, Aiuti A, Ambrosi A, Biasco L, Bondanza A, Lambiase A, Traversari C, Vago L, von Kalle C, Schmidt M, Bordignon C, Ciceri F, Bonini C.

Sci Transl Med. 2015 Dec 9;7(317):317ra198. doi: 10.1126/scitranslmed.aac8265. Erratum in: Sci Transl Med. 2015 Dec 23;7(319):319er9. D'Agostino, Mattio [corrected to D'Agostino, Mattia].

PMID:
26659572
46.

Combined immunodeficiency due to JAK3 mutation in a child presenting with skin granuloma.

Scarselli A, Di Cesare S, Di Matteo G, De Matteis A, Ariganello P, Romiti ML, Cascioli S, De Vito R, Bertaina A, Locatelli F, Gaspar HB, Aiuti A, Rossi P, Gilmour K, Cancrini C.

J Allergy Clin Immunol. 2016 Mar;137(3):948-51.e5. doi: 10.1016/j.jaci.2015.09.017. Epub 2015 Nov 3. No abstract available.

PMID:
26545580
47.

Lentiviral-mediated gene therapy restores B cell tolerance in Wiskott-Aldrich syndrome patients.

Pala F, Morbach H, Castiello MC, Schickel JN, Scaramuzza S, Chamberlain N, Cassani B, Glauzy S, Romberg N, Candotti F, Aiuti A, Bosticardo M, Villa A, Meffre E.

J Clin Invest. 2015 Oct 1;125(10):3941-51. doi: 10.1172/JCI82249. Epub 2015 Sep 14.

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Autoimmunity and regulatory T cells in 22q11.2 deletion syndrome patients.

Di Cesare S, Puliafito P, Ariganello P, Marcovecchio GE, Mandolesi M, Capolino R, Digilio MC, Aiuti A, Rossi P, Cancrini C.

Pediatr Allergy Immunol. 2015 Sep;26(6):591-4. doi: 10.1111/pai.12420. No abstract available.

PMID:
26058917
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Longitudinal Evaluation of Immune Reconstitution and B-cell Function After Hematopoietic Cell Transplantation for Primary Immunodeficiency.

Scarselli A, Di Cesare S, Capponi C, Cascioli S, Romiti ML, Di Matteo G, Simonetti A, Palma P, Finocchi A, Lucarelli B, Pinto RM, Rana I, Palumbo G, Caniglia M, Rossi P, Carsetti R, Cancrini C, Aiuti A.

J Clin Immunol. 2015 May;35(4):373-83. doi: 10.1007/s10875-015-0154-4. Epub 2015 Apr 15.

PMID:
25875698
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Clinical applications of gene therapy for primary immunodeficiencies.

Cicalese MP, Aiuti A.

Hum Gene Ther. 2015 Apr;26(4):210-9. doi: 10.1089/hum.2015.047. Review.

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