相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges
Marina Cavazzana et al.
NATURE REVIEWS DRUG DISCOVERY (2019)
Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease
Laura I. Marquez Loza et al.
GENES (2019)
GBA, Gaucher Disease, and Parkinson's Disease: From Genetic to Clinic to New Therapeutic Approaches
Giulietta M. Riboldi et al.
CELLS (2019)
A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments
Jerome Stirnemann et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2017)
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Florian Eichler et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes
Eric Sherman et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)
INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions
Charles C. Berry et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)
Simultaneous quantitation of sphingoid bases by UPLC-ESI-MS/MS with identical 13C-encoded internal standards
M. Mirzaian et al.
CLINICA CHIMICA ACTA (2017)
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
Mario Sessa et al.
LANCET (2016)
The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease
Carla E. M. Hollak et al.
BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM (2015)
Lentiviral Gene Therapy Using Cellular Promoters Cures Type 1 Gaucher Disease in Mice
Maria Dahl et al.
MOLECULAR THERAPY (2015)
Gaucher disease: haematological presentations and complications
Alison S. Thomas et al.
BRITISH JOURNAL OF HAEMATOLOGY (2014)
Gaucher Disease and Bone Manifestations
Gemma Marcucci et al.
CALCIFIED TISSUE INTERNATIONAL (2014)
Hemorrhagic Aspects of Gaucher Disease
Hanna Rosenbaum
RAMBAM MAIMONIDES MEDICAL JOURNAL (2014)
Altered Composition of Bone as Triggered by Irradiation Facilitates the Rapid Erosion of the Matrix by Both Cellular and Physicochemical Processes
Danielle E. Green et al.
PLOS ONE (2013)
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
Alessandra Biffi et al.
SCIENCE (2013)
Epidermal growth factor improves lentivirus vector gene transfer into primary mouse hepatocytes
M. Rothe et al.
GENE THERAPY (2012)
Risk factors for fractures and avascular osteonecrosis in type 1 Gaucher disease: A study from the International Collaborative Gaucher Group (ICGG) Gaucher Registry
Aneal Khan et al.
JOURNAL OF BONE AND MINERAL RESEARCH (2012)
The French Gaucher's disease registry: clinical characteristics, complications and treatment of 562 patients
Jerome Stirnemann et al.
ORPHANET JOURNAL OF RARE DISEASES (2012)
Long-term complications and side effects after allogeneic hematopoietic stem cell transplantation: an update
B. Mohty et al.
BLOOD CANCER JOURNAL (2011)
Bone density in type 1 gaucher disease
Gregory M. Pastores et al.
JOURNAL OF BONE AND MINERAL RESEARCH (2010)
Total-Body Irradiation of Postpubertal Mice with 137Cs Acutely Compromises the Microarchitecture of Cancellous Bone and Increases Osteoclasts
Hisataka Kondo et al.
RADIATION RESEARCH (2009)
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Nathalie Cartier et al.
SCIENCE (2009)
Successful Low-Risk Hematopoietic Cell Therapy in a Mouse Model of Type 1 Gaucher Disease
Ida Berglin Enquist et al.
STEM CELLS (2009)
Lysosomal storage disease 1 - Phenotype, diagnosis, and treatment of Gaucher's disease
Gregory A. Grabowski
LANCET (2008)
The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease
J. Charrow et al.
CLINICAL GENETICS (2007)
Effective cell and gene therapy in a murine model of Gaucher disease
Ida Berglin Enquist et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
A murine model for bone loss from therapeutic and space-relevant sources of radiation
S. A. Hamilton et al.
JOURNAL OF APPLIED PHYSIOLOGY (2006)
Species comparison of postnatal bone growth and development
T Zoetis et al.
BIRTH DEFECTS RESEARCH PART B-DEVELOPMENTAL AND REPRODUCTIVE TOXICOLOGY (2003)
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: A report from the Gaucher Registry
NJ Weinreb et al.
AMERICAN JOURNAL OF MEDICINE (2002)
Skeletal aspects of Gaucher disease: a review
RJ Wenstrup et al.
BRITISH JOURNAL OF RADIOLOGY (2002)
分享论文
