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Items: 37

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.

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.

4.

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
5.

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.

6.

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.

7.

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
8.

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.

9.

NADPH Oxidase Deficiency: A Multisystem Approach.

Giardino G, Cicalese MP, Delmonte O, Migliavacca M, Palterer B, Loffredo L, Cirillo E, Gallo V, Violi F, Pignata C.

Oxid Med Cell Longev. 2017;2017:4590127. doi: 10.1155/2017/4590127. Epub 2017 Dec 21. Review.

10.

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.

11.

Beta2 integrins are required for follicular helper T cell differentiation in humans.

Gerosa J, Lougaris V, Baronio M, Plebani A, Cicalese MP, Fousteri G.

Clin Immunol. 2017 Jul;180:60-62. doi: 10.1016/j.clim.2017.04.002. Epub 2017 Apr 6. No abstract available.

PMID:
28390967
12.

Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy.

Cossu G, Previtali SC, Napolitano S, Cicalese MP, Tedesco FS, Nicastro F, Noviello M, Roostalu U, Natali Sora MG, Scarlato M, De Pellegrin M, Godi C, Giuliani S, Ciotti F, Tonlorenzi R, Lorenzetti I, Rivellini C, Benedetti S, Gatti R, Marktel S, Mazzi B, Tettamanti A, Ragazzi M, Imro MA, Marano G, Ambrosi A, Fiori R, Sormani MP, Bonini C, Venturini M, Politi LS, Torrente Y, Ciceri F.

EMBO Mol Med. 2016 Dec 1;8(12):1470-1471. doi: 10.15252/emmm.201607129. No abstract available.

13.

Deconstructing IgG4-related disease involvement of midline structures: Comparison to common mimickers.

Lanzillotta M, Campochiaro C, Trimarchi M, Arrigoni G, Gerevini S, Milani R, Bozzolo E, Biafora M, Venturini E, Cicalese MP, Stone JH, Sabbadini MG, Della-Torre E.

Mod Rheumatol. 2017 Jul;27(4):638-645. doi: 10.1080/14397595.2016.1227026. Epub 2016 Sep 13.

PMID:
27622319
14.

Longitudinal MRI quantification of muscle degeneration in Duchenne muscular dystrophy.

Godi C, Ambrosi A, Nicastro F, Previtali SC, Santarosa C, Napolitano S, Iadanza A, Scarlato M, Natali Sora MG, Tettamanti A, Gerevini S, Cicalese MP, Sitzia C, Venturini M, Falini A, Gatti R, Ciceri F, Cossu G, Torrente Y, Politi LS.

Ann Clin Transl Neurol. 2016 Jun 16;3(8):607-22. doi: 10.1002/acn3.319. eCollection 2016 Aug.

15.

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
16.

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.

17.

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.

18.

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.

19.

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.

20.

Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy.

Cossu G, Previtali SC, Napolitano S, Cicalese MP, Tedesco FS, Nicastro F, Noviello M, Roostalu U, Natali Sora MG, Scarlato M, De Pellegrin M, Godi C, Giuliani S, Ciotti F, Tonlorenzi R, Lorenzetti I, Rivellini C, Benedetti S, Gatti R, Marktel S, Mazzi B, Tettamanti A, Ragazzi M, Imro MA, Marano G, Ambrosi A, Fiori R, Sormani MP, Bonini C, Venturini M, Politi LS, Torrente Y, Ciceri F.

EMBO Mol Med. 2015 Dec;7(12):1513-28. doi: 10.15252/emmm.201505636. Erratum in: EMBO Mol Med. 2016 Dec 1;8(12 ):1470-1471.

21.

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.

22.

Abnormalities of acid-base balance and predisposition to metabolic acidosis in Metachromatic Leukodystrophy patients.

Lorioli L, Cicalese MP, Silvani P, Assanelli A, Salvo I, Mandelli A, Fumagalli F, Fiori R, Ciceri F, Aiuti A, Sessa M, Roncarolo MG, Lanzani C, Biffi A.

Mol Genet Metab. 2015 May;115(1):48-52. doi: 10.1016/j.ymgme.2015.02.009. Epub 2015 Mar 6.

PMID:
25796965
23.

Central venous access devices in pediatric malignancies: a position paper of Italian Association of Pediatric Hematology and Oncology.

Crocoli A, Tornesello A, Pittiruti M, Barone A, Muggeo P, Inserra A, Molinari AC, Grillenzoni V, Durante V, Cicalese MP, Zanazzo GA, Cesaro S.

J Vasc Access. 2015 Mar-Apr;16(2):130-6. doi: 10.5301/jva.5000314. Epub 2014 Oct 14. Review.

PMID:
25362978
24.

Successful use of long-acting octreotide for intractable chronic gastrointestinal bleeding in children.

O'Meara M, Cicalese MP, Bordugo A, Ambrosi A, Hadzic N, Mieli-Vergani G.

J Pediatr Gastroenterol Nutr. 2015 Jan;60(1):48-53. doi: 10.1097/MPG.0000000000000540.

PMID:
25162361
25.

Inflammation converts human mesoangioblasts into targets of alloreactive immune responses: implications for allogeneic cell therapy of DMD.

Noviello M, Tedesco FS, Bondanza A, Tonlorenzi R, Rosaria Carbone M, Gerli MFM, Marktel S, Napolitano S, Cicalese MP, Ciceri F, Peretti G, Cossu G, Bonini C.

Mol Ther. 2014 Jul;22(7):1342-1352. doi: 10.1038/mt.2014.62. Epub 2014 Apr 16.

26.

B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients.

Brigida I, Sauer AV, Ferrua F, Giannelli S, Scaramuzza S, Pistoia V, Castiello MC, Barendregt BH, Cicalese MP, Casiraghi M, Brombin C, Puck J, Müller K, Notarangelo LD, Montin D, van Montfrans JM, Roncarolo MG, Traggiai E, van Dongen JJ, van der Burg M, Aiuti A.

J Allergy Clin Immunol. 2014 Mar;133(3):799-806.e10. doi: 10.1016/j.jaci.2013.12.1043. Epub 2014 Feb 5.

27.

Intergenerational and intrafamilial phenotypic variability in 22q11.2 deletion syndrome subjects.

Cirillo E, Giardino G, Gallo V, Puliafito P, Azzari C, Bacchetta R, Cardinale F, Cicalese MP, Consolini R, Martino S, Martire B, Molinatto C, Plebani A, Scarano G, Soresina A, Cancrini C, Rossi P, Digilio MC, Pignata C.

BMC Med Genet. 2014 Jan 2;15:1. doi: 10.1186/1471-2350-15-1.

28.

Single-day trimethoprim/sulfamethoxazole prophylaxis for Pneumocystis pneumonia in children with cancer.

Caselli D, Petris MG, Rondelli R, Carraro F, Colombini A, Muggeo P, Ziino O, Melchionda F, Russo G, Pierani P, Soncini E, DeSantis R, Zanazzo G, Barone A, Cesaro S, Cellini M, Mura R, Milano GM, Meazza C, Cicalese MP, Tropia S, De Masi S, Castagnola E, Aricò M; Infectious Diseases Working Group of the Associazione Italiana Ematologia Oncologia Pediatrica.

J Pediatr. 2014 Feb;164(2):389-92.e1. doi: 10.1016/j.jpeds.2013.10.021. Epub 2013 Nov 16.

PMID:
24252793
29.

Risk of seizures in children receiving busulphan-containing regimens for stem cell transplantation.

Caselli D, Rosati A, Faraci M, Podda M, Ripaldi M, Longoni D, Cesaro S, Lo Nigro L, Paolicchi O, Maximova N, Menconi MC, Ziino O, Cicalese MP, Santarone S, Nesi F, Aricò M, Locatelli F, Prete A; Bone Marrow Transplantation Working Group of the Associazione Italiana Ematologia Oncologia Pediatrica.

Biol Blood Marrow Transplant. 2014 Feb;20(2):282-5. doi: 10.1016/j.bbmt.2013.10.028. Epub 2013 Nov 4.

30.

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, Zanetti G, Rizzo WB, Mehta NA, Cicalese MP, Casiraghi M, Boelens JJ, Del Carro U, Dow DJ, Schmidt M, Assanelli A, Neduva V, Di Serio C, Stupka E, Gardner J, von Kalle C, Bordignon C, Ciceri F, Rovelli A, Roncarolo MG, Aiuti A, Sessa M, Naldini L.

Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11.

31.

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, Dionisio F, Calabria A, Giannelli S, Castiello MC, Bosticardo M, Evangelio C, Assanelli A, Casiraghi M, Di Nunzio S, Callegaro L, Benati C, Rizzardi P, Pellin D, Di Serio C, Schmidt M, Von Kalle C, Gardner J, Mehta N, Neduva V, Dow DJ, Galy A, Miniero R, Finocchi A, Metin A, Banerjee PP, Orange JS, Galimberti S, Valsecchi MG, Biffi A, Montini E, Villa A, Ciceri F, Roncarolo MG, Naldini L.

Science. 2013 Aug 23;341(6148):1233151. doi: 10.1126/science.1233151. Epub 2013 Jul 11.

32.

Guidelines for the use of long-term central venous catheter in children with hemato-oncological disorders. On behalf of supportive therapy working group of Italian Association of Pediatric Hematology and Oncology (AIEOP).

Carraro F, Cicalese MP, Cesaro S, De Santis R, Zanazzo G, Tornesello A, Giordano P, Bergadano A, Giacchino M.

Ann Hematol. 2013 Oct;92(10):1405-12. doi: 10.1007/s00277-013-1794-1. Epub 2013 Jun 16.

PMID:
23771477
33.

Human IL2RA null mutation mediates immunodeficiency with lymphoproliferation and autoimmunity.

Goudy K, Aydin D, Barzaghi F, Gambineri E, Vignoli M, Ciullini Mannurita S, Doglioni C, Ponzoni M, Cicalese MP, Assanelli A, Tommasini A, Brigida I, Dellepiane RM, Martino S, Olek S, Aiuti A, Ciceri F, Roncarolo MG, Bacchetta R.

Clin Immunol. 2013 Mar;146(3):248-61. doi: 10.1016/j.clim.2013.01.004. Epub 2013 Jan 24.

34.

Management of autoimmune hepatitis in children: how many steps away from common agreement?

Cicalese MP, Iorio R.

J Pediatr Gastroenterol Nutr. 2012 Oct;55(4):364. No abstract available.

PMID:
22644465
35.

Purine metabolism, immune reconstitution, and abdominal adipose tumor after gene therapy for adenosine deaminase deficiency.

Grunebaum E, Chung CT, Dadi H, Kim P, Brigida I, Ferrua F, Cicalese MP, Aiuti A, Roifman CM.

J Allergy Clin Immunol. 2011 Jun;127(6):1417-9.e3. doi: 10.1016/j.jaci.2011.04.014. Epub 2011 Apr 29. No abstract available.

PMID:
21531016
36.

Relationship between severe obesity and gut inflammation in children: what's next?

Spagnuolo MI, Cicalese MP, Caiazzo MA, Franzese A, Squeglia V, Assante LR, Valerio G, Merone R, Guarino A.

Ital J Pediatr. 2010 Oct 1;36:66. doi: 10.1186/1824-7288-36-66.

37.

Requesting iodine supplementation in children on parenteral nutrition.

Cicalese MP, Bruzzese E, Guarino A, Spagnuolo MI.

Clin Nutr. 2009 Jun;28(3):256-9. doi: 10.1016/j.clnu.2009.02.005. Epub 2009 Mar 9.

PMID:
19269721

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