Marcella Facchini
Overview
Explore the profile of Marcella Facchini including associated specialties, affiliations and a list of published articles.
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20
Citations
691
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Recent Articles
1.
Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, et al.
Sci Transl Med
. 2024 May;
16(745):eadi8214.
PMID: 38691622
Mucopolysaccharidosis type I Hurler (MPSIH) is characterized by severe and progressive skeletal dysplasia that is not fully addressed by allogeneic hematopoietic stem cell transplantation (HSCT). Autologous hematopoietic stem progenitor cell-gene...
2.
Fumagalli F, Calbi V, Sora M, Sessa M, Baldoli C, Rancoita P, et al.
Lancet
. 2022 Jan;
399(10322):372-383.
PMID: 35065785
Background: Effective treatment for metachromatic leukodystrophy (MLD) remains a substantial unmet medical need. In this study we investigated the safety and efficacy of atidarsagene autotemcel (arsa-cel) in patients with MLD....
3.
Gentner B, Tucci F, Galimberti S, Fumagalli F, De Pellegrin M, Silvani P, et al.
N Engl J Med
. 2021 Nov;
385(21):1929-1940.
PMID: 34788506
Background: Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with...
4.
Fumagalli F, Zambon A, Rancoita P, Baldoli C, Canale S, Spiga I, et al.
J Inherit Metab Dis
. 2021 Apr;
44(5):1151-1164.
PMID: 33855715
In this study, we characterize the natural course of metachromatic leukodystrophy (MLD), explore intra/inter group differences, and identify biomarkers to monitor disease progression. This is a longitudinal observational study. Genotype...
5.
Tucci F, Frittoli M, Barzaghi F, Calbi V, Migliavacca M, Ferrua F, et al.
Bone Marrow Transplant
. 2019 Jun;
54(12):1995-2003.
PMID: 31150018
Collection of an adequate amount of autologous haematopoietic stem progenitor cells (HSPC) is required for ex vivo manipulation and successful engraftment for certain inherited disorders. Fifty-seven paediatric patients (age 0.5-11.4...
6.
Ferrua F, Cicalese M, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, et al.
Lancet Haematol
. 2019 Apr;
6(5):e239-e253.
PMID: 30981783
Background: Wiskott-Aldrich syndrome is a rare, life-threatening, X-linked primary immunodeficiency characterised by microthrombocytopenia, infections, eczema, autoimmunity, and malignant disease. Lentiviral vector-mediated haemopoietic stem/progenitor cell (HSPC) gene therapy is a potentially...
7.
Targeting the Bacterial Cytoskeleton of the Complex for Antimicrobial Development: A Cautionary Tale
Carnell S, Perry J, Borthwick L, Vollmer D, Biboy J, Facchini M, et al.
Int J Mol Sci
. 2018 Jun;
19(6).
PMID: 29848957
complex (BCC) bacteria are a group of opportunistic pathogens that cause severe lung infections in cystic fibrosis (CF). Treatment of BCC infections is difficult, due to the inherent and acquired...
8.
Cigana C, Bernardini F, Facchini M, Alcala-Franco B, Riva C, De Fino I, et al.
Antimicrob Agents Chemother
. 2016 Jun;
60(8):4991-5000.
PMID: 27297477
The clinical development of antibiotics with a new mode of action combined with efficient pulmonary drug delivery is a priority against untreatable Pseudomonas aeruginosa lung infections. POL7001 is a macrocycle...
9.
Minandri F, Imperi F, Frangipani E, Bonchi C, Visaggio D, Facchini M, et al.
Infect Immun
. 2016 Jun;
84(8):2324-2335.
PMID: 27271740
Pseudomonas aeruginosa is a leading cause of hospital-acquired pneumonia and chronic lung infections in cystic fibrosis patients. Iron is essential for bacterial growth, and P. aeruginosa expresses multiple iron uptake...
10.
Bianconi I, Jeukens J, Freschi L, Alcala-Franco B, Facchini M, Boyle B, et al.
BMC Genomics
. 2015 Dec;
16:1105.
PMID: 26714629
Background: Pseudomonas aeruginosa establishes life-long chronic airway infections in cystic fibrosis (CF) patients. As the disease progresses, P. aeruginosa pathoadaptive variants are distinguished from the initially acquired strain. However, the...