Exploiting the Potential of Drosophila Models in Lysosomal Storage Disorders: Pathological Mechanisms and Drug Discovery

Blood–Brain Barrier Dynamics to Maintain Brain Homeostasis

(2021) Marta Segarra et al.   TRENDS IN NEUROSCIENCES

Mucopolysaccharidosis Type II: One Hundred Years of Research, Diagnosis, and Treatment

(2020) Francesca D’Avanzo et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Pathogenesis of Mucopolysaccharidoses, an Update

(2020) Simona Fecarotta et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

The Drosophila septate junctions beyond barrier function: Review of the literature, prediction of human orthologs of the SJ‐related proteins and identification of protein domain families

(2020) Erasmia Rouka et al.   Acta Physiologica

Dysfunction of the Blood-Brain Barrier—A Key Step in Neurodegeneration and Dementia

(2020) Christian R. Noe et al.   Frontiers in Aging Neuroscience

Organelle Cooperation in Stem Cell Fate: Lysosomes as Emerging Regulators of Cell Identity

(2020) Lisa M. Julian et al.   Frontiers in Cell and Developmental Biology

Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

(2020) Jacob M. Favret et al.   Frontiers in Molecular Biosciences

Modeling Lysosomal Storage Diseases in the Zebrafish

(2020) T. Zhang et al.   Frontiers in Molecular Biosciences

Misfolding of Lysosomal α-Galactosidase a in a Fly Model and Its Alleviation by the Pharmacological Chaperone Migalastat

(2020) Hila Braunstein et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Neuroinflammation in neurodegenerative disorders: the roles of microglia and astrocytes

(2020) Hyuk Sung Kwon et al.   Translational Neurodegeneration

The effect of mutant GBA1 on accumulation and aggregation of α-synuclein

(2019) Gali Maor et al.   HUMAN MOLECULAR GENETICS

Lysosomal storage disorders – challenges, concepts and avenues for therapy: beyond rare diseases

(2019) André R. A. Marques et al.   JOURNAL OF CELL SCIENCE

Migalastat: A Review in Fabry Disease

(2019) Emma H. McCafferty et al.   DRUGS

Flavonoids Regulate Lipid Droplets Biogenesis in Drosophila melanogaster

(2019) Marianna Fantin et al.   Natural Product Communications

Arylsulfatase A, a genetic modifier of Parkinson’s disease, is an α-synuclein chaperone

(2019) Jun Sung Lee et al.   BRAIN

Live applications of norbormide-based fluorescent probes in Drosophila melanogaster

(2019) Alessia Forgiarini et al.   PLoS One

Regulation of the Blood–Brain Barrier by Circadian Rhythms and Sleep

(2019) Vishnu Anand Cuddapah et al.   TRENDS IN NEUROSCIENCES

A triple drug combination targeting components of the nutrient-sensing network maximizes longevity

(2019) Jorge Iván Castillo-Quan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Drosophila melanogaster Mutated in its GBA1b Ortholog Recapitulates Neuronopathic Gaucher Disease

(2019) Cabasso et al.   Journal of Clinical Medicine

Lysosomes as dynamic regulators of cell and organismal homeostasis

(2019) Andrea Ballabio et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Glial Phagocytic Receptors Promote Neuronal Loss in Adult Drosophila Brain

(2019) Ketty Hakim-Mishnaevski et al.   Cell Reports

A Drosophila model of neuronal ceroid lipofuscinosis CLN4 reveals a hypermorphic gain of function mechanism

(2019) Elliot Imler et al.   eLife

Naringenin Ameliorates Drosophila ReepA Hereditary Spastic Paraplegia-Linked Phenotypes

(2019) Barbara Napoli et al.   Frontiers in Neuroscience

Drosophila Rab2 controls endosome-lysosome fusion and LAMP delivery to late endosomes

(2018) Viktor Karlovich Lund et al.   Autophagy

Autophagy in health and disease: A comprehensive review

(2018) Sarbari Saha et al.   BIOMEDICINE & PHARMACOTHERAPY

A Circadian Clock in the Blood-Brain Barrier Regulates Xenobiotic Efflux

(2018) Shirley L. Zhang et al.   CELL

Glycophagy: An emerging target in pathology

(2018) Hong Zhao et al.   CLINICA CHIMICA ACTA

Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA

(2018) Dani L. Webber et al.   EXPERIMENTAL NEUROLOGY

Bridging barriers: a comparative look at the blood–brain barrier across organisms

(2018) Natasha M. O'Brown et al.   GENES & DEVELOPMENT

Inborn Errors of Metabolism Involving Complex Molecules

(2018) Cinzia Maria Bellettato et al.   PEDIATRIC CLINICS OF NORTH AMERICA

Whole-Cell Scale Dynamic Organization of Lysosomes Revealed by Spatial Statistical Analysis

(2018) Qinle Ba et al.   Cell Reports

High Throughput Measurement of Locomotor Sensitization to Volatilized Cocaine in Drosophila melanogaster

(2018) Ana Filošević et al.   Frontiers in Molecular Neuroscience

Drosophila melanogaster as a function-based high-throughput screening model for anti-nephrolithiasis agents in kidney stone patients

(2018) Sohrab N. Ali et al.   Disease Models & Mechanisms

Lysosome biogenesis in health and disease

(2018) Lakshya Bajaj et al.   JOURNAL OF NEUROCHEMISTRY

Measurement of solid food intake in Drosophila via consumption-excretion of a dye tracer

(2018) Brandon C. Shell et al.   Scientific Reports

Origin of the Autophagosome Membrane in Mammals

(2018) Yun Wei et al.   Biomed Research International

Neuromuscular degeneration and locomotor deficit in a Drosophila model of mucopolysaccharidosis VII is attenuated by treatment with resveratrol

(2018) Sudipta Bar et al.   Disease Models & Mechanisms

The lysosome as a cellular centre for signalling, metabolism and quality control

(2018) Rosalie E. Lawrence et al.   NATURE CELL BIOLOGY

Activated Microglia Disrupt the Blood-Brain Barrier and Induce Chemokines and Cytokines in a Rat in vitro Model

(2018) Yukari Shigemoto-Mogami et al.   Frontiers in Cellular Neuroscience

Lysosomal storage diseases

(2018) Frances M. Platt et al.   Nature Reviews Disease Primers

Dysregulation of autophagy as a common mechanism in lysosomal storage diseases

(2017) Elena Seranova et al.   Essays in Biochemistry

Minos-insertion mutant of the Drosophila GBA gene homologue showed abnormal phenotypes of climbing ability, sleep and life span with accumulation of hydroxy-glucocerebroside

(2017) Haruhisa Kawasaki et al.   GENE

Gustatory receptor 22e is essential for sensing chloroquine and strychnine in Drosophila melanogaster

(2017) Seeta Poudel et al.   INSECT BIOCHEMISTRY AND MOLECULAR BIOLOGY

Mdr65 decreases toxicity of multiple insecticides in Drosophila melanogaster

(2017) Haina Sun et al.   INSECT BIOCHEMISTRY AND MOLECULAR BIOLOGY

Lipid metabolic perturbation is an early-onset phenotype in adult spinster mutants: a Drosophila model for lysosomal storage disorders

(2017) Sarita Hebbar et al.   MOLECULAR BIOLOGY OF THE CELL

Epidemiology of mucopolysaccharidoses

(2017) Shaukat A. Khan et al.   MOLECULAR GENETICS AND METABOLISM

A saposin deficiency model in Drosophila : Lysosomal storage, progressive neurodegeneration and sensory physiological decline

(2017) Samantha J. Hindle et al.   NEUROBIOLOGY OF DISEASE

Emerging Roles for the Lysosome in Lipid Metabolism

(2017) Ashley M. Thelen et al.   TRENDS IN CELL BIOLOGY

Comparative studies of vertebrate iduronate 2-sulfatase (IDS) genes and proteins: evolution of A mammalian X-linked gene

(2017) Roger S. Holmes   3 Biotech

Comparative studies of vertebrate iduronate 2-sulfatase (IDS) genes and proteins: evolution of A mammalian X-linked gene

(2017) Roger S. Holmes   3 Biotech

Exploring Autophagy in Drosophila

(2017) Péter Lőrincz et al.   Cells

The Lysosome as a Regulatory Hub

(2016) Rushika M. Perera et al.   Annual Review of Cell and Developmental Biology

A functional endosomal pathway is necessary for lysosome biogenesis in Drosophila

(2016) Anne-Claire Jacomin et al.   BMC CELL BIOLOGY

Modeling congenital disease and inborn errors of development inDrosophila melanogaster

(2016) Matthew J. Moulton et al.   Disease Models & Mechanisms

The translational relevance ofDrosophilain drug discovery

(2016) Ismael Fernández‐Hernández et al.   EMBO REPORTS

Mechanisms and functions of lysosome positioning

(2016) Jing Pu et al.   JOURNAL OF CELL SCIENCE

A Drosophila Model of Neuronopathic Gaucher Disease Demonstrates Lysosomal-Autophagic Defects and Altered mTOR Signalling and Is Functionally Rescued by Rapamycin

(2016) Kerri J. Kinghorn et al.   JOURNAL OF NEUROSCIENCE

Cancer Drug Development Using Drosophila as an in vivo Tool: From Bedside to Bench and Back

(2016) Amarish Kumar Yadav et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Glucocerebrosidase Deficiency in Drosophila Results in α-Synuclein-Independent Protein Aggregation and Neurodegeneration

(2016) Marie Y. Davis et al.   PLoS Genetics

An antibody toolkit for the study of membrane traffic inDrosophila melanogaster

(2016) Falko Riedel et al.   Biology Open

An integrated platform for large-scale data collection and precise perturbation of live Drosophila embryos

(2016) Thomas J. Levario et al.   Scientific Reports

Neuronal ceroid lipofuscinosis with DNAJC5/CSPα mutation has PPT1 pathology and exhibit aberrant protein palmitoylation

(2015) Michael X. Henderson et al.   ACTA NEUROPATHOLOGICA

Syntaxin1A-mediated Resistance and Hypersensitivity to Isoflurane in Drosophila melanogaster

(2015) Oressia H. Zalucki et al.   ANESTHESIOLOGY

Epidemiology and diagnosis of lysosomal storage disorders; challenges of screening

(2015) Sandra D.K. Kingma et al.   BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM

Metachromatic leukodystrophy: Disease spectrum and approaches for treatment

(2015) Diane F. van Rappard et al.   BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM

The mucolipidosis IV Ca2+ channel TRPML1 (MCOLN1) is regulated by the TOR kinase

(2015) R. U. Onyenwoke et al.   BIOCHEMICAL JOURNAL

Glial Lipid Droplets and ROS Induced by Mitochondrial Defects Promote Neurodegeneration

(2015) Lucy Liu et al.   CELL

A Century of Cholesterol and Coronaries: From Plaques to Genes to Statins

(2015) Joseph L. Goldstein et al.   CELL

Recycling endosomes

(2015) James R Goldenring   CURRENT OPINION IN CELL BIOLOGY

Genetics on the Fly: A Primer on the Drosophila Model System

(2015) K. G. Hales et al.   GENETICS

Gaucher Disease: Clinical, Biological and Therapeutic Aspects

(2015) Azza Dandana et al.   PATHOBIOLOGY

Diminished MTORC1-Dependent JNK Activation Underlies the Neurodevelopmental Defects Associated with Lysosomal Dysfunction

(2015) Ching-On Wong et al.   Cell Reports

Systematic Evaluation of Drosophila CRISPR Tools Reveals Safe and Robust Alternatives to Autonomous Gene Drives in Basic Research

(2015) Fillip Port et al.   G3-Genes Genomes Genetics

Gap Junction Proteins in the Blood-Brain Barrier Control Nutrient-Dependent Reactivation of Drosophila Neural Stem Cells

(2014) Pauline Spéder et al.   DEVELOPMENTAL CELL

Glycogen pathways in disease: new developments in a classical field of medical genetics

(2014) Manfred W. Kilimann et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Quantifying Drosophila food intake: comparative analysis of current methodology

(2014) Sonali A Deshpande et al.   NATURE METHODS

Electron spin changes during general anesthesia in Drosophila

(2014) L. Turin et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Optimized CRISPR/Cas tools for efficient germline and somatic genome engineering in Drosophila

(2014) F. Port et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Autophagy inDrosophila: From Historical Studies to Current Knowledge

(2014) Nitha C. Mulakkal et al.   Biomed Research International

The Drosophila surface glia transcriptome: evolutionary conserved blood-brain barrier processes

(2014) Michael K. DeSalvo et al.   Frontiers in Neuroscience

The Drosophila blood-brain barrier: development and function of a glial endothelium

(2014) Stefanie Limmer et al.   Frontiers in Neuroscience

Animal models for lysosomal storage disorders

(2013) G. M. Pastores et al.   BIOCHEMISTRY-MOSCOW

A method for reversible drug delivery to internal tissues ofDrosophilaembryos

(2013) Victoria K Schulman et al.   FLY

Innexins Ogre and Inx2 are required in glial cells for normal postembryonic development of the Drosophila central nervous system

(2013) C. E. Holcroft et al.   JOURNAL OF CELL SCIENCE

Development, maintenance and disruption of the blood-brain barrier

(2013) Birgit Obermeier et al.   NATURE MEDICINE

Unfolded protein response in Gaucher disease: from human to Drosophila

(2013) Gali Maor et al.   Orphanet Journal of Rare Diseases

Expression of Human Gaucher Disease Gene GBA Generates Neurodevelopmental Defects and ER Stress in Drosophila Eye

(2013) Takahiro Suzuki et al.   PLoS One

High Resolution Crystal Structure of Human β-Glucuronidase Reveals Structural Basis of Lysosome Targeting

(2013) Md. Imtaiyaz Hassan et al.   PLoS One

The lysosome: from waste bag to potential therapeutic target

(2013) Hanna Appelqvist et al.   Journal of Molecular Cell Biology

Acid sensing by sweet and bitter taste neurons in Drosophila melanogaster

(2013) Sandhya Charlu et al.   Nature Communications

Small molecule drug screening in Drosophila identifies the 5HT2A receptor as a feeding modulation target

(2013) Gabriel Gasque et al.   Scientific Reports

Role of Autophagy in Glycogen Breakdown and Its Relevance to Chloroquine Myopathy

(2013) Jonathan Zirin et al.   PLOS BIOLOGY

Lipid metabolism in Drosophila: development and disease

(2012) Z. Liu et al.   ACTA BIOCHIMICA ET BIOPHYSICA SINICA

The neuronal ceroid-lipofuscinoses: A historical introduction

(2012) Matti Haltia et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Drosophila TRPML Is Required for TORC1 Activation

(2012) Ching-On Wong et al.   CURRENT BIOLOGY

An in vivo large-scale chemical screening platform using Drosophila for anti-cancer drug discovery

(2012) L. F. Willoughby et al.   Disease Models & Mechanisms

Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age

(2012) F. Matthes et al.   HUMAN MOLECULAR GENETICS

Rab5 is necessary for the biogenesis of the endolysosomal system in vivo

(2012) Anja Zeigerer et al.   NATURE

Glycolytic oligodendrocytes maintain myelin and long-term axonal integrity

(2012) Ursula Fünfschilling et al.   NATURE

FlyAtlas: database of gene expression in the tissues of Drosophila melanogaster

(2012) S. W. Robinson et al.   NUCLEIC ACIDS RESEARCH

Chaperone-mediated autophagy: a unique way to enter the lysosome world

(2012) Susmita Kaushik et al.   TRENDS IN CELL BIOLOGY

Mutations in DNAJC5, Encoding Cysteine-String Protein Alpha, Cause Autosomal-Dominant Adult-Onset Neuronal Ceroid Lipofuscinosis

(2011) Lenka Nosková et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Dendrimer D5 is a Vector for Peptide Transport to Brain Cells

(2011) S. V. Sarantseva et al.   BULLETIN OF EXPERIMENTAL BIOLOGY AND MEDICINE

Microautophagy: lesser-known self-eating

(2011) Wen-wen Li et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Endosome maturation

(2011) Jatta Huotari et al.   EMBO JOURNAL

The Batten disease gene CLN3 is required for the response to oxidative stress

(2011) Richard I. Tuxworth et al.   HUMAN MOLECULAR GENETICS

Invertebrate models of lysosomal storage disease: what have we learned so far?

(2011) Samantha Hindle et al.   INVERTEBRATE NEUROSCIENCE

Sweet Sixteen for ANLS

(2011) Luc Pellerin et al.   JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM

Human Disease Models in Drosophila melanogaster and the Role of the Fly in Therapeutic Drug Discovery

(2011) U. B. Pandey et al.   PHARMACOLOGICAL REVIEWS

Spinster is required for autophagic lysosome reformation and mTOR reactivation following starvation

(2011) Y. Rong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Lysosomal membrane proteins: life between acid and neutral conditions: Figure 1

(2010) Paul Saftig et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

High-throughput screening and small animal models, where are we?

(2010) Jean Giacomotto et al.   BRITISH JOURNAL OF PHARMACOLOGY

Micellar capillary electrophoresis - Electrochemical detection of neurochemicals fromDrosophila

(2010) Nicholas J. Kuklinski et al.   JOURNAL OF SEPARATION SCIENCE

Termination of autophagy and reformation of lysosomes regulated by mTOR

(2010) Li Yu et al.   NATURE

The Batten Disease Palmitoyl Protein Thioesterase 1 Gene Regulates Neural Specification and Axon Connectivity during Drosophila Embryonic Development

(2010) Quynh Chu-LaGraff et al.   PLoS One

Towards understanding the neuronal ceroid lipofuscinoses

(2009) Alfried Kohlschütter et al.   BRAIN & DEVELOPMENT

Neuroprotective Role of Lactate after Cerebral Ischemia

(2009) Carole Berthet et al.   JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM

Ensheathing Glia Function as Phagocytes in the Adult Drosophila Brain

(2009) J. Doherty et al.   JOURNAL OF NEUROSCIENCE

Lysosome biogenesis and lysosomal membrane proteins: trafficking meets function

(2009) Paul Saftig et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Genetic modifiers of degeneration in the cathepsin D deficient Drosophila model for neuronal ceroid lipofuscinosis

(2009) Mervi Kuronen et al.   NEUROBIOLOGY OF DISEASE

A Gene Network Regulating Lysosomal Biogenesis and Function

(2009) Marco Sardiello et al.   SCIENCE

Disorders of Lysosome-Related Organelle Biogenesis: Clinical and Molecular Genetics

(2008) Marjan Huizing et al.   Annual Review of Genomics and Human Genetics

Motor Deficit in a Drosophila Model of Mucolipidosis Type IV due to Defective Clearance of Apoptotic Cells

(2008) Kartik Venkatachalam et al.   CELL

Interactions between the juvenile Batten disease gene, CLN3, and the Notch and JNK signalling pathways

(2008) Richard I. Tuxworth et al.   HUMAN MOLECULAR GENETICS

Neuronal Loss of Drosophila NPC1a Causes Cholesterol Aggregation and Age-Progressive Neurodegeneration

(2008) S. E. Phillips et al.   JOURNAL OF NEUROSCIENCE

Organization and Function of the Blood Brain Barrier in Drosophila

(2008) T. Stork et al.   JOURNAL OF NEUROSCIENCE

Chemically modified  -glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII

(2008) J. H. Grubb et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Elevated globotriaosylsphingosine is a hallmark of Fabry disease

(2008) J. M. Aerts et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA