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Items: 1 to 20 of 53

1.

Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients.

Esrick EB, Manis JP, Daley H, Baricordi C, Trébéden-Negre H, Pierciey FJ, Armant M, Nikiforow S, Heeney MM, London WB, Biasco L, Asmal M, Williams DA, Biffi A.

Blood Adv. 2018 Oct 9;2(19):2505-2512. doi: 10.1182/bloodadvances.2018016725.

2.

Prostaglandin E2 Stimulates the Expansion of Regulatory Hematopoietic Stem and Progenitor Cells in Type 1 Diabetes.

Ben Nasr M, D'Addio F, Malvandi AM, Faravelli S, Castillo-Leon E, Usuelli V, Rocchio F, Letizia T, El Essawy AB, Assi E, Mameli C, Giani E, Macedoni M, Maestroni A, Dassano A, Loretelli C, Paroni M, Cannalire G, Biasucci G, Sala M, Biffi A, Zuccotti GV, Fiorina P.

Front Immunol. 2018 Jun 19;9:1387. doi: 10.3389/fimmu.2018.01387. eCollection 2018.

3.

Metachromatic Leukodystrophy: Too Frequent (Mis)Diagnosis?

Politi LS, Salsano E, Biffi A.

JAMA Neurol. 2018 Aug 1;75(8):1027. doi: 10.1001/jamaneurol.2018.1515. No abstract available.

PMID:
29913002
4.

Hydrazone linked doxorubicin-PLA prodrug nanoparticles with high drug loading.

Gatti S, Agostini A, Palmiero UC, Colombo C, Peviani M, Biffi A, Moscatelli D.

Nanotechnology. 2018 Jul 27;29(30):305602. doi: 10.1088/1361-6528/aac0d3. Epub 2018 Apr 27.

PMID:
29701613
5.

Gene Therapy as a Curative Option for β-Thalassemia.

Biffi A.

N Engl J Med. 2018 Apr 19;378(16):1551-1552. doi: 10.1056/NEJMe1802169. No abstract available.

PMID:
29669229
6.

Metallothioneins are neuroprotective agents in lysosomal storage disorders.

Cavalca E, Cesani M, Gifford JC, Sena-Esteves M, Terreni MR, Leoncini G, Peviani M, Biffi A.

Ann Neurol. 2018 Feb;83(2):418-432. doi: 10.1002/ana.25161. Epub 2018 Feb 15.

PMID:
29369397
7.

Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells.

Capotondo A, Milazzo R, Garcia-Manteiga JM, Cavalca E, Montepeloso A, Garrison BS, Peviani M, Rossi DJ, Biffi A.

Sci Adv. 2017 Dec 6;3(12):e1701211. doi: 10.1126/sciadv.1701211. eCollection 2017 Dec.

8.

Microglial dysfunction as a key pathological change in adrenomyeloneuropathy.

Gong Y, Sasidharan N, Laheji F, Frosch M, Musolino P, Tanzi R, Kim DY, Biffi A, El Khoury J, Eichler F.

Ann Neurol. 2017 Nov;82(5):813-827. doi: 10.1002/ana.25085. Epub 2017 Nov 11.

9.

Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases.

Biffi A.

Hematol Oncol Clin North Am. 2017 Oct;31(5):869-881. doi: 10.1016/j.hoc.2017.06.004. Epub 2017 Jul 23. Review.

PMID:
28895853
10.

Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.

Biffi A.

Mol Ther. 2017 May 3;25(5):1155-1162. doi: 10.1016/j.ymthe.2017.03.025. Epub 2017 Apr 4. Review.

11.

Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies.

Visigalli I, Delai S, Ferro F, Cecere F, Vezzoli M, Sanvito F, Chanut F, Benedicenti F, Spinozzi G, Wynn R, Calabria A, Naldini L, Montini E, Cristofori P, Biffi A.

Hum Gene Ther. 2016 Oct;27(10):813-829. doi: 10.1089/hum.2016.068.

PMID:
27431943
12.

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
13.

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.

14.

Voices of biotech.

Amit I, Baker D, Barker R, Berger B, Bertozzi C, Bhatia S, Biffi A, Demichelis F, Doudna J, Dowdy SF, Endy D, Helmstaedter M, Junca H, June C, Kamb S, Khvorova A, Kim DH, Kim JS, Krishnan Y, Lakadamyali M, Lappalainen T, Lewin S, Liao J, Loman N, Lundberg E, Lynd L, Martin C, Mellman I, Miyawaki A, Mummery C, Nelson K, Paz J, Peralta-Yahya P, Picotti P, Polyak K, Prather K, Qin J, Quake S, Regev A, Rogers JA, Shetty R, Sommer M, Stevens M, Stolovitzky G, Takahashi M, Tang F, Teichmann S, Torres-Padilla ME, Tripathi L, Vemula P, Verdine G, Vollmer F, Wang J, Ying JY, Zhang F, Zhang T.

Nat Biotechnol. 2016 Mar;34(3):270-5. doi: 10.1038/nbt.3502. No abstract available.

PMID:
26963549
15.

Gene therapy for lysosomal storage disorders: a good start.

Biffi A.

Hum Mol Genet. 2016 Apr 15;25(R1):R65-75. doi: 10.1093/hmg/ddv457. Epub 2015 Nov 24. Review.

PMID:
26604151
16.

Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy.

Ungari S, Montepeloso A, Morena F, Cocchiarella F, Recchia A, Martino S, Gentner B, Naldini L, Biffi A.

Mol Ther Methods Clin Dev. 2015 Oct 14;2:15038. doi: 10.1038/mtm.2015.38. eCollection 2015.

17.

Mutation Update of ARSA and PSAP Genes Causing Metachromatic Leukodystrophy.

Cesani M, Lorioli L, Grossi S, Amico G, Fumagalli F, Spiga I, Filocamo M, Biffi A.

Hum Mutat. 2016 Jan;37(1):16-27. doi: 10.1002/humu.22919. Epub 2015 Nov 4. Review.

PMID:
26462614
18.

Mesoangioblast delivery of miniagrin ameliorates murine model of merosin-deficient congenital muscular dystrophy type 1A.

Domi T, Porrello E, Velardo D, Capotondo A, Biffi A, Tonlorenzi R, Amadio S, Ambrosi A, Miyagoe-Suzuki Y, Takeda S, Ruegg MA, Previtali SC.

Skelet Muscle. 2015 Sep 3;5:30. doi: 10.1186/s13395-015-0055-5. eCollection 2015.

19.

Clinical translation of TALENS: Treating SCID-X1 by gene editing in iPSCs.

Biffi A.

Cell Stem Cell. 2015 Apr 2;16(4):348-9. doi: 10.1016/j.stem.2015.03.009.

20.

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.

Ricca A, Rufo N, Ungari S, Morena F, Martino S, Kulik W, Alberizzi V, Bolino A, Bianchi F, Del Carro U, Biffi A, Gritti A.

Hum Mol Genet. 2015 Jun 15;24(12):3372-89. doi: 10.1093/hmg/ddv086. Epub 2015 Mar 5.

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