Related references
Note: Only part of the references are listed.A Brief History of Protein Sorting Prediction
Henrik Nielsen et al.
PROTEIN JOURNAL (2019)
Targeted delivery of lysosomal enzymes to the endocytic compartment in human cells using engineered extracellular vesicles
Mai Anh Do et al.
SCIENTIFIC REPORTS (2019)
CDD/SPARCLE: functional classification of proteins via subfamily domain architectures
Aron Marchler-Bauer et al.
NUCLEIC ACIDS RESEARCH (2017)
Lysosomal Storage Diseases: From Pathophysiology to Therapy
Giancarlo Parenti et al.
ANNUAL REVIEW OF MEDICINE, VOL 66 (2015)
Development and Applications of CRISPR-Cas9 for Genome Engineering
Patrick D. Hsu et al.
CELL (2014)
Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library
Hiroko Koike-Yusa et al.
NATURE BIOTECHNOLOGY (2014)
Genome engineering: the next genomic revolution
Charles A. Gersbach
NATURE METHODS (2014)
A TALEN-based strategy for efficient bi-allelic miRNA ablation in human cells
Claudia Uhde-Stone et al.
RNA (2014)
Genome Engineering at the Dawn of the Golden Age
David J. Segal et al.
ANNUAL REVIEW OF GENOMICS AND HUMAN GENETICS, VOL 14 (2013)
Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
Seung Woo Cho et al.
NATURE BIOTECHNOLOGY (2013)
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
Thomas Gaj et al.
TRENDS IN BIOTECHNOLOGY (2013)
Animal models for Gaucher disease research
Tamar Farfel-Becker et al.
DISEASE MODELS & MECHANISMS (2011)
Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals
Raymond Y. Wang et al.
GENETICS IN MEDICINE (2011)
Global gene disruption in human cells to assign genes to phenotypes by deep sequencing
Jan E. Carette et al.
NATURE BIOTECHNOLOGY (2011)
Enzyme replacement therapy for Gaucher disease
Joel Charrow
EXPERT OPINION ON BIOLOGICAL THERAPY (2009)
Haploid Genetic Screens in Human Cells Identify Host Factors Used by Pathogens
Jan E. Carette et al.
SCIENCE (2009)
Gaucher disease:: Mutation and polymorphism spectrum in the glucocerebrosidase gene (GBA)
Kathleen S. Hruska et al.
HUMAN MUTATION (2008)
Lysosomal storage disease 1 - Phenotype, diagnosis, and treatment of Gaucher's disease
Gregory A. Grabowski
LANCET (2008)
Enzyme reconstitution/replacement therapy for lysosomal storage diseases
T. Andrew Burrow et al.
CURRENT OPINION IN PEDIATRICS (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)
Limitations of enzyme replacement therapy: Current and future
J. E. Wraith
JOURNAL OF INHERITED METABOLIC DISEASE (2006)
The cell biology of lysosomal storage disorders
AH Futerman et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2004)