Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 1 to 50 of 197

1.

Biological Properties of HSC: Scientific Basis for HSCT.

Aiuti A, Scala S, Chabannon C.

In: Carreras E, Dufour C, Mohty M, Kröger N, editors. The EBMT Handbook: Hematopoietic Stem Cell Transplantation and Cellular Therapies [Internet]. 7th edition. Cham (CH): Springer; 2019. Chapter 7.

2.

New perspectives in gene therapy for inherited disorders.

Cicalese MP, Aiuti A.

Pediatr Allergy Immunol. 2020 Feb;31 Suppl 24:5-7. doi: 10.1111/pai.13149.

PMID:
32017225
3.

A Prevalent CXCR3+ Phenotype of Circulating Follicular Helper T Cells Indicates Humoral Dysregulation in Children with Down Syndrome.

Ottaviano G, Gerosa J, Santini M, De Leo P, Vecchione A, Jofra T, Trimarchi C, De Pellegrin M, Agosti M, Aiuti A, Marinoni M, Cicalese MP, Fousteri G.

J Clin Immunol. 2020 Jan 28. doi: 10.1007/s10875-020-00755-0. [Epub ahead of print]

PMID:
31993866
4.

Expanded circulating hematopoietic stem/progenitor cells as novel cell source for the treatment of TCIRG1 osteopetrosis.

Capo V, Penna S, Merelli I, Barcella M, Scala S, Basso-Ricci L, Draghici E, Palagano E, Zonari E, Desantis G, Uva P, Cusano R, Sergi Sergi L, Crisafulli L, Moshous D, Stepensky P, Drabko K, Kaya Z, Unal E, Gezdirici A, Menna G, Serafini M, Aiuti A, Locatelli SL, Carlo-Stella C, Schulz AS, Ficara F, Sobacchi C, Gentner B, Villa A.

Haematologica. 2020 Jan 16. pii: haematol.2019.238261. doi: 10.3324/haematol.2019.238261. [Epub ahead of print]

5.

Leukocyte and Dried Blood Spot Arylsulfatase A Assay by Tandem Mass Spectrometry.

Hong X, Kumar AB, Daiker J, Yi F, Sadilek M, De Mattia F, Fumagalli F, Calbi V, Damiano R, Bona MD, la Marca G, Vanderver AL, Waldman AT, Adang L, Sherbini O, Woidill S, Suhr T, Kurtzberg J, Beltran-Quintero ML, Escolar M, Aiuti A, Gelb MH.

Anal Chem. 2020 Jan 10. doi: 10.1021/acs.analchem.9b05274. [Epub ahead of print]

PMID:
31922725
6.

Treatment with rapamycin can restore regulatory T-cell function in IPEX patients.

Passerini L, Barzaghi F, Curto R, Sartirana C, Barera G, Tucci F, Albarello L, Mariani A, Testoni PA, Bazzigaluppi E, Bosi E, Lampasona V, Neth O, Zama D, Hoenig M, Schulz A, Seidel MG, Rabbone I, Olek S, Roncarolo MG, Cicalese MP, Aiuti A, Bacchetta R.

J Allergy Clin Immunol. 2019 Dec 23. pii: S0091-6749(19)32592-8. doi: 10.1016/j.jaci.2019.11.043. [Epub ahead of print]

PMID:
31874182
7.

Health-Related Quality of Life and Emotional Difficulties in Chronic Granulomatous Disease: Data on Adult and Pediatric Patients from Italian Network for Primary Immunodeficiency (IPINet).

Pulvirenti F, Sangerardi M, Plebani A, Soresina A, Finocchi A, Pignata C, Cirillo E, Trizzino A, Aiuti A, Migliavacca M, Locatelli F, Bertaina A, Naviglio S, Carrabba M, De Carli M, Barbaro MGF, Gattorno M, Quinti I, Martire B.

J Clin Immunol. 2020 Feb;40(2):289-298. doi: 10.1007/s10875-019-00725-1. Epub 2019 Dec 20.

PMID:
31863244
8.

Reduced PD-1 expression on circulating follicular and conventional FOXP3+ Treg cells in children with new onset type 1 diabetes and autoantibody-positive at-risk children.

Vecchione A, Di Fonte R, Gerosa J, Jofra T, Cicalese MP, Napoleone V, Ippolito E, Galvani G, Ragogna F, Stabilini A, Bianconi E, Grogan P, Bonura C, Bonfanti R, Frontino G, Nano R, Melzi R, De Pellegrin M, Laurenzi A, Meschi F, Barera G, Rigamonti A, Indirli R, Bosi E, Piemonti L, Aiuti A, Battaglia M, Fousteri G.

Clin Immunol. 2020 Feb;211:108319. doi: 10.1016/j.clim.2019.108319. Epub 2019 Nov 30.

PMID:
31794865
9.

NFKB2 regulates human Tfh and Tfr pool formation and germinal center potential.

De Leo P, Gazzurelli L, Baronio M, Montin D, Di Cesare S, Giancotta C, Licciardi F, Cancrini C, Aiuti A, Plebani A, Cicalese MP, Lougaris V, Fousteri G.

Clin Immunol. 2020 Jan;210:108309. doi: 10.1016/j.clim.2019.108309. Epub 2019 Nov 18.

PMID:
31751612
10.

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives.

Staal FJT, Aiuti A, Cavazzana M.

Front Pediatr. 2019 Oct 31;7:443. doi: 10.3389/fped.2019.00443. eCollection 2019. Review.

11.

A novel disorder involving dyshematopoiesis, inflammation, and HLH due to aberrant CDC42 function.

Lam MT, Coppola S, Krumbach OHF, Prencipe G, Insalaco A, Cifaldi C, Brigida I, Zara E, Scala S, Di Cesare S, Martinelli S, Di Rocco M, Pascarella A, Niceta M, Pantaleoni F, Ciolfi A, Netter P, Carisey AF, Diehl M, Akbarzadeh M, Conti F, Merli P, Pastore A, Levi Mortera S, Camerini S, Farina L, Buchholzer M, Pannone L, Cao TN, Coban-Akdemir ZH, Jhangiani SN, Muzny DM, Gibbs RA, Basso-Ricci L, Chiriaco M, Dvorsky R, Putignani L, Carsetti R, Janning P, Stray-Pedersen A, Erichsen HC, Horne A, Bryceson YT, Torralba-Raga L, Ramme K, Rosti V, Bracaglia C, Messia V, Palma P, Finocchi A, Locatelli F, Chinn IK, Lupski JR, Mace EM, Cancrini C, Aiuti A, Ahmadian MR, Orange JS, De Benedetti F, Tartaglia M.

J Exp Med. 2019 Dec 2;216(12):2778-2799. doi: 10.1084/jem.20190147. Epub 2019 Oct 10.

PMID:
31601675
12.

Clinical, Immunological, and Molecular Features of Typical and Atypical Severe Combined Immunodeficiency: Report of the Italian Primary Immunodeficiency Network.

Cirillo E, Cancrini C, Azzari C, Martino S, Martire B, Pession A, Tommasini A, Naviglio S, Finocchi A, Consolini R, Pierani P, D'Alba I, Putti MC, Marzollo A, Giardino G, Prencipe R, Esposito F, Grasso F, Scarselli A, Di Matteo G, Attardi E, Ricci S, Montin D, Specchia F, Barzaghi F, Cicalese MP, Quaremba G, Lougaris V, Giliani S, Locatelli F, Rossi P, Aiuti A, Badolato R, Plebani A, Pignata C.

Front Immunol. 2019 Aug 13;10:1908. doi: 10.3389/fimmu.2019.01908. eCollection 2019.

13.

In vivo dynamics of human hematopoietic stem cells: novel concepts and future directions.

Scala S, Aiuti A.

Blood Adv. 2019 Jun 25;3(12):1916-1924. doi: 10.1182/bloodadvances.2019000039. Review.

14.

Corrigendum: Targeted NGS Platforms for Genetic Screening and Gene Discovery in Primary Immunodeficiencies.

Cifaldi C, Brigida I, Barzaghi F, Zoccolillo M, Ferradini V, Petricone D, Cicalese MP, Lazarevic D, Cittaro D, Omrani M, Attardi E, Conti F, Scarselli A, Chiriaco M, Di Cesare S, Licciardi F, Davide M, Ferrua F, Canessa C, Pignata C, Giliani S, Ferrari S, Fousteri G, Barera G, Merli P, Palma P, Cesaro S, Gattorno M, Trizzino A, Moschese V, Chini L, Villa A, Azzari C, Finocchi A, Locatelli F, Rossi P, Sangiuolo F, Aiuti A, Cancrini C, Di Matteo G.

Front Immunol. 2019 May 31;10:1184. doi: 10.3389/fimmu.2019.01184. eCollection 2019.

15.

Advances in stem cell research and therapeutic development.

De Luca M, Aiuti A, Cossu G, Parmar M, Pellegrini G, Robey PG.

Nat Cell Biol. 2019 Jul;21(7):801-811. doi: 10.1038/s41556-019-0344-z. Epub 2019 Jun 17. Review.

PMID:
31209293
16.

Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.

Tucci F, Frittoli M, Barzaghi F, Calbi V, Migliavacca M, Ferrua F, Fumagalli F, Lorioli L, Castagnaro L, Facchini M, Fossati C, Zancan S, Massariello P, Manfredini M, Consiglieri G, Canarutto D, Recupero S, Calzatini F, Casiraghi M, Darin S, Antonioli G, Miniero R, Fiori R, Silvani P, Zambelli M, Marktel S, Gattillo S, Milani R, Santoleri L, Ciceri F, Biffi A, Cicalese MP, Bernardo ME, Aiuti A.

Bone Marrow Transplant. 2019 Dec;54(12):1995-2003. doi: 10.1038/s41409-019-0573-6. Epub 2019 May 31.

17.

Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease.

Squeri G, Passerini L, Ferro F, Laudisa C, Tomasoni D, Deodato F, Donati MA, Gasperini S, Aiuti A, Bernardo ME, Gentner B, Naldini L, Annoni A, Biffi A, Gregori S.

Mol Ther. 2019 Jul 3;27(7):1215-1227. doi: 10.1016/j.ymthe.2019.04.014. Epub 2019 Apr 19.

18.

Targeted NGS Platforms for Genetic Screening and Gene Discovery in Primary Immunodeficiencies.

Cifaldi C, Brigida I, Barzaghi F, Zoccolillo M, Ferradini V, Petricone D, Cicalese MP, Lazarevic D, Cittaro D, Omrani M, Attardi E, Conti F, Scarselli A, Chiriaco M, Di Cesare S, Licciardi F, Davide M, Ferrua F, Canessa C, Pignata C, Giliani S, Ferrari S, Fousteri G, Barera G, Merli P, Palma P, Cesaro S, Gattorno M, Trizzino A, Moschese V, Chini L, Villa A, Azzari C, Finocchi A, Locatelli F, Rossi P, Sangiuolo F, Aiuti A, Cancrini C, Di Matteo G.

Front Immunol. 2019 Apr 11;10:316. doi: 10.3389/fimmu.2019.00316. eCollection 2019. Erratum in: Front Immunol. 2019 May 31;10:1184.

19.

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 MP, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo ME, Calbi V, Assanelli AA, Facchini M, Fossati C, Albertazzi E, Scaramuzza S, Brigida I, Scala S, Basso-Ricci L, Pajno R, Casiraghi M, Canarutto D, Salerio FA, Albert MH, Bartoli A, Wolf HM, Fiori R, Silvani P, Gattillo S, Villa A, Biasco L, Dott C, Culme-Seymour EJ, van Rossem K, Atkinson G, Valsecchi MG, Roncarolo MG, Ciceri F, Naldini L, Aiuti A.

Lancet Haematol. 2019 May;6(5):e239-e253. doi: 10.1016/S2352-3026(19)30021-3. Epub 2019 Apr 10.

20.

Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome.

Sereni L, Castiello MC, Di Silvestre D, Della Valle P, Brombin C, Ferrua F, Cicalese MP, Pozzi L, Migliavacca M, Bernardo ME, Pignata C, Farah R, Notarangelo LD, Marcus N, Cattaneo L, Spinelli M, Giannelli S, Bosticardo M, van Rossem K, D'Angelo A, Aiuti A, Mauri P, Villa A.

J Allergy Clin Immunol. 2019 Sep;144(3):825-838. doi: 10.1016/j.jaci.2019.03.012. Epub 2019 Mar 27.

21.

First Case of Patient With Two Homozygous Mutations in MYD88 and CARD9 Genes Presenting With Pyogenic Bacterial Infections, Elevated IgE, and Persistent EBV Viremia.

Chiriaco M, Di Matteo G, Conti F, Petricone D, De Luca M, Di Cesare S, Cifaldi C, De Vito R, Zoccolillo M, Serafinelli J, Poerio N, Fraziano M, Brigida I, Cardinale F, Rossi P, Aiuti A, Cancrini C, Finocchi A.

Front Immunol. 2019 Feb 14;10:130. doi: 10.3389/fimmu.2019.00130. eCollection 2019.

22.

Bone marrow stromal cells from β-thalassemia patients have impaired hematopoietic supportive capacity.

Crippa S, Rossella V, Aprile A, Silvestri L, Rivis S, Scaramuzza S, Pirroni S, Avanzini MA, Basso-Ricci L, Hernandez RJ, Zecca M, Marktel S, Ciceri F, Aiuti A, Ferrari G, Bernardo ME.

J Clin Invest. 2019 Feb 25;129(4):1566-1580. doi: 10.1172/JCI123191. eCollection 2019 Feb 25.

23.

A combined immunodeficiency with severe infections, inflammation, and allergy caused by ARPC1B deficiency.

Volpi S, Cicalese MP, Tuijnenburg P, Tool ATJ, Cuadrado E, Abu-Halaweh M, Ahanchian H, Alzyoud R, Akdemir ZC, Barzaghi F, Blank A, Boisson B, Bottino C, Brigida I, Caorsi R, Casanova JL, Chiesa S, Chinn IK, Dückers G, Enders A, Erichsen HC, Forbes LR, Gambin T, Gattorno M, Karimiani EG, Giliani S, Gold MS, Jacobsen EM, Jansen MH, King JR, Laxer RM, Lupski JR, Mace E, Marcenaro S, Maroofian R, Meijer AB, Niehues T, Notarangelo LD, Orange J, Pannicke U, Pearson C, Picco P, Quinn PJ, Schulz A, Seeborg F, Stray-Pedersen A, Tawamie H, van Leeuwen EMM, Aiuti A, Yeung R, Schwarz K, Kuijpers TW.

J Allergy Clin Immunol. 2019 Jun;143(6):2296-2299. doi: 10.1016/j.jaci.2019.02.003. Epub 2019 Feb 13. No abstract available.

24.

ALPS-Like Phenotype Caused by ADA2 Deficiency Rescued by Allogeneic Hematopoietic Stem Cell Transplantation.

Barzaghi F, Minniti F, Mauro M, Bortoli M, Balter R, Bonetti E, Zaccaron A, Vitale V, Omrani M, Zoccolillo M, Brigida I, Cicalese MP, Degano M, Hershfield MS, Aiuti A, Bondarenko AV, Chinello M, Cesaro S.

Front Immunol. 2019 Jan 14;9:2767. doi: 10.3389/fimmu.2018.02767. eCollection 2018.

25.

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.

Marktel S, Scaramuzza S, Cicalese MP, Giglio F, Galimberti S, Lidonnici MR, Calbi V, Assanelli A, Bernardo ME, Rossi C, Calabria A, Milani R, Gattillo S, Benedicenti F, Spinozzi G, Aprile A, Bergami A, Casiraghi M, Consiglieri G, Masera N, D'Angelo E, Mirra N, Origa R, Tartaglione I, Perrotta S, Winter R, Coppola M, Viarengo G, Santoleri L, Graziadei G, Gabaldo M, Valsecchi MG, Montini E, Naldini L, Cappellini MD, Ciceri F, Aiuti A, Ferrari G.

Nat Med. 2019 Feb;25(2):234-241. doi: 10.1038/s41591-018-0301-6. Epub 2019 Jan 21.

PMID:
30664781
26.

Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches.

Fraldi A, Serafini M, Sorrentino NC, Gentner B, Aiuti A, Bernardo ME.

Ital J Pediatr. 2018 Nov 16;44(Suppl 2):130. doi: 10.1186/s13052-018-0565-y. Review.

27.

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 Nov;24(11):1683-1690. doi: 10.1038/s41591-018-0195-3. Epub 2018 Oct 1.

PMID:
30275570
28.

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, 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 Nov 29;132(22):2362-2374. doi: 10.1182/blood-2018-07-863431. Epub 2018 Sep 25.

29.

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 Nov;194(2):166-179. doi: 10.1111/cei.13163. Epub 2018 Sep 24. Review.

30.

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. 2019 Mar;143(3):852-863. doi: 10.1016/j.jaci.2018.08.024. Epub 2018 Sep 5. Review.

31.

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.

32.

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. 2019 May;41(4):e266-e269. doi: 10.1097/MPH.0000000000001256.

PMID:
30044346
33.

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.

34.

Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV 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, Abd Elaziz D, Ciceri F, Aiuti A, Cicalese MP.

Hepatology. 2018 Dec;68(6):2434-2437. doi: 10.1002/hep.30160. Epub 2018 Nov 13. No abstract available.

35.

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.

36.

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.

37.

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. Review.

38.

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 Nov;142(5):1605-1617.e4. doi: 10.1016/j.jaci.2017.11.063. Epub 2018 Feb 13.

39.

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.

40.

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.

41.

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
42.

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.

43.

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.

44.

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
45.

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
46.

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.

47.

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.

48.

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.

49.

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
50.

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.

Supplemental Content

Support Center