Giuliana Ferrari

università vita-salute san raffaele

H指数:51

被引数:1.3万

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The withdrawal of beti-cel therapy for β-Thalassemia from the European market: a multistakeholder perspective

β-地中海贫血beti-cel疗法退出欧洲市场的多利益相关方视角

Orphanet Journal of Rare Diseases

IF3.5

2026-09-040

Claudio Bordignon

Molecular Therapy

IF12

2026-06-160

PRE

Iron overload damages mitochondria and induces metabolic rewiring of hematopoietic stem cells towards glycolysis

铁过载损伤线粒体，并诱导造血干细胞向糖酵解方向进行代谢重编程。

Blood

IF23.1

2026-03-260

OAAI

Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies

β0-地中海贫血突变的碱基编辑作为一种治疗严重β-血红蛋白病的策略

Science Translational Medicine

IF14.6

2025-11-260

PRE

Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche

血红蛋白病基因治疗：临床试验结果及造血干细胞生物学与骨髓微环境

Cell Reports Medicine

IF10.6

2025-11-170

OAAI

Immunomodulatory effects of dental pulp stem cells on lymphocytes and monocytes from patients with rheumatoid arthritis

牙髓干细胞对类风湿关节炎患者淋巴细胞和单核细胞的免疫调节作用

CLINICAL AND EXPERIMENTAL RHEUMATOLOGY

IF3.3

2025-05-080

PRE

Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells

MOLECULAR THERAPY

IF12

2024-01-012

OAAI

A single approach to targeting transferrin receptor 2 corrects iron and erythropoietic defects in murine models of anemia of inflammation and chronic disease

KIDNEY INTERNATIONAL

IF12.6

2023-07-0111

PRE

Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

结合基因添加和基因沉默策略的镰状细胞病基因治疗的新型慢病毒载体

MOLECULAR THERAPY NUCLEIC ACIDS

IF6.1

2023-06-015

OAAI

Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia

SCIENCE TRANSLATIONAL MEDICINE

IF14.6

2023-05-314

PRE

Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy

基因工程满足下一代基因治疗的造血干细胞生物学

CELL STEM CELL

IF20.4

2023-05-0122

Hypomorphic mutation of the TALE gene Prep1 (pKnox1) causes a major reduction of Pbx and Meis proteins and a pleiotropic embryonic phenotype

MOLECULAR AND CELLULAR BIOLOGY

IF2.7

2023-03-2795

OAAI

Assessing and enhancing migration of human myogenic progenitors using directed iPS cell differentiation and advanced tissue modelling

EMBO MOLECULAR MEDICINE

IF8.3

2022-09-2610

OAAI

Transferrin receptor 2 (Tfr2) genetic deletion makes transfusion-independent a murine model of transfusion-dependent β-thalassemia

AMERICAN JOURNAL OF HEMATOLOGY

IF9.9

2022-08-109

Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease

靶向 β-地中海贫血和镰状细胞病中的造血干细胞生态位

PHARMACEUTICALS

IF4.8

2022-05-115

OAAI

Investigating the role of dystrophin isoform deficiency in motor function in Duchenne muscular dystrophy

JOURNAL OF CACHEXIA SARCOPENIA AND MUSCLE

IF9.1

2022-01-2627

OAAI

Dystrophin deficiency affects human astrocyte properties andresponse to damage

肌营养不良蛋白缺乏症会影响人类星形胶质细胞的特性和对损伤的反应

GLIA

IF5.1

2021-11-1315

OAAI

Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling

MOLECULAR THERAPY-NUCLEIC ACIDS

IF0

2021-03-0111

OAAI

Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

通过CRISPR/Cas9编辑人类造血干细胞中的 α-珠蛋白基因座来校正 β-地中海贫血

BLOOD ADVANCES

IF7.1

2021-02-2655

Gene therapy using haematopoietic stem and progenitor cells

使用造血干细胞和祖细胞的基因治疗

NATURE REVIEWS GENETICS

IF52

2020-12-10160

OAAI

Giuliana Ferrari

个人简介

教育背景

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