Intrabodies as Neuroprotective Therapeutics

Nanobodies Raised against Monomeric α-Synuclein Distinguish between Fibrils at Different Maturation Stages

(2013) Tim Guilliams et al.   JOURNAL OF MOLECULAR BIOLOGY

α-synuclein levels affect autophagosome numbers in vivo and modulate Huntington disease pathology

(2012) Silvia Corrochano et al.   Autophagy

RNA-binding proteins in microsatellite expansion disorders: Mediators of RNA toxicity

(2012) Gloria V. Echeverria et al.   BRAIN RESEARCH

Physical Chemistry of Polyglutamine: Intriguing Tales of a Monotonous Sequence

(2012) Ronald Wetzel   JOURNAL OF MOLECULAR BIOLOGY

Fusion to a highly charged proteasomal retargeting sequence increases soluble cytoplasmic expression and efficacy of diverse anti-synuclein intrabodies

(2012) Shubhada N. Joshi et al.   mAbs

Alpha-Synuclein Posttranslational Modification and Alternative Splicing as a Trigger for Neurodegeneration

(2012) Katrin Beyer et al.   MOLECULAR NEUROBIOLOGY

Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses

(2012) Agnieszka Ciesielska et al.   MOLECULAR THERAPY

“Huntingtin Holiday”: Progress toward an Antisense Therapy for Huntington's Disease

(2012) Xiao-Hong Lu et al.   NEURON

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

Acid β-glucosidase mutants linked to gaucher disease, parkinson disease, and lewy body dementia alter α-synuclein processing

(2011) Valerie Cullen et al.   ANNALS OF NEUROLOGY

Parkinson's Disease: Genetics and Pathogenesis

(2011) Joshua M. Shulman et al.   Annual Review of Pathology-Mechanisms of Disease

α-Synuclein modifies huntingtin aggregation in living cells

(2011) Federico Herrera et al.   FEBS LETTERS

Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

(2011) James A. Allay et al.   HUMAN GENE THERAPY

α-Synuclein accumulates in huntingtin inclusions but forms independent filaments and its deficiency attenuates early phenotype in a mouse model of Huntington's disease

(2011) Cristina Tomás-Zapico et al.   HUMAN MOLECULAR GENETICS

Genetic therapy for the nervous system

(2011) W. J. Bowers et al.   HUMAN MOLECULAR GENETICS

Protein Misfolding Detected Early in Pathogenesis of Transgenic Mouse Model of Huntington Disease Using Amyloid Seeding Assay

(2011) Sharad Gupta et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

The Rate of PolyQ-Mediated Aggregation Is Dramatically Affected by the Number and Location of Surrounding Domains

(2011) Amy L. Robertson et al.   JOURNAL OF MOLECULAR BIOLOGY

A Disulfide-Free Single-Domain VL Intrabody with Blocking Activity towards Huntingtin Reveals a Novel Mode of Epitope Recognition

(2011) André Schiefner et al.   JOURNAL OF MOLECULAR BIOLOGY

Imputation of sequence variants for identification of genetic risks for Parkinson's disease: a meta-analysis of genome-wide association studies

(2011)   LANCET

Leucine-rich repeat kinase 2 and alpha-synuclein: intersecting pathways in the pathogenesis of Parkinson's disease?

(2011) Elisa Greggio et al.   Molecular Neurodegeneration

Priorities in Parkinson's disease research

(2011) Wassilios G. Meissner et al.   NATURE REVIEWS DRUG DISCOVERY

Gene therapy for Huntington's disease

(2011) Shilpa Ramaswamy et al.   NEUROBIOLOGY OF DISEASE

Huntington's Disease: Can Mice Lead the Way to Treatment?

(2011) Zachary R. Crook et al.   NEURON

Perturbation with Intrabodies Reveals That Calpain Cleavage Is Required for Degradation of Huntingtin Exon 1

(2011) Amber L. Southwell et al.   PLoS One

Bifunctional Anti-Huntingtin Proteasome-Directed Intrabodies Mediate Efficient Degradation of Mutant Huntingtin Exon 1 Protein Fragments

(2011) David C. Butler et al.   PLoS One

Engineered antibody therapies to counteract mutant huntingtin and related toxic intracellular proteins

(2011) David C. Butler et al.   PROGRESS IN NEUROBIOLOGY

A Therapeutic Antibody Targeting BACE1 Inhibits Amyloid-  Production in Vivo

(2011) J. K. Atwal et al.   Science Translational Medicine

Boosting Brain Uptake of a Therapeutic Antibody by Reducing Its Affinity for a Transcytosis Target

(2011) Y. J. Yu et al.   Science Translational Medicine

Potential cellular and regenerative approaches for the treatment of Parkinson’s disease

(2011) Emma Lane et al.   Neuropsychiatric Disease and Treatment

Rilmenidine attenuates toxicity of polyglutamine expansions in a mouse model of Huntington's disease

(2010) C. Rose et al.   HUMAN MOLECULAR GENETICS

Structure and Properties of a Complex of α-Synuclein and a Single-Domain Camelid Antibody

(2010) Erwin J. De Genst et al.   JOURNAL OF MOLECULAR BIOLOGY

Early or Late-Stage Anti-N-Terminal Huntingtin Intrabody Gene Therapy Reduces Pathological Features in B6.HDR6/1 Mice

(2010) Abigail Snyder-Keller et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Harnessing chaperone-mediated autophagy for the selective degradation of mutant huntingtin protein

(2010) Peter O Bauer et al.   NATURE BIOTECHNOLOGY

Cystamine and intrabody co-treatment confers additional benefits in a fly model of Huntington's disease

(2010) S.F. Bortvedt et al.   NEUROBIOLOGY OF DISEASE

Nicotinamide improves motor deficits and upregulates PGC-1α and BDNF gene expression in a mouse model of Huntington's disease

(2010) Tyisha Hathorn et al.   NEUROBIOLOGY OF DISEASE

Increased Expression of α-Synuclein Reduces Neurotransmitter Release by Inhibiting Synaptic Vesicle Reclustering after Endocytosis

(2010) Venu M. Nemani et al.   NEURON

Loss of leucine-rich repeat kinase 2 causes impairment of protein degradation pathways, accumulation of  -synuclein, and apoptotic cell death in aged mice

(2010) Y. Tong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Non-ATG-initiated translation directed by microsatellite expansions

(2010) T. Zu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Physico-chemical determinants of soluble intrabody expression in mammalian cell cytoplasm

(2010) Erik Kvam et al.   PROTEIN ENGINEERING DESIGN & SELECTION

Recombinant Fv-Hsp70 Protein Mediates Neuroprotection After Focal Cerebral Ischemia in Rats

(2010) Xinhua Zhan et al.   STROKE

1H, 13C and 15N assignments of a camelid nanobody directed against human α-synuclein

(2009) Anneleen Vuchelen et al.   Biomolecular NMR Assignments

Developing intrabodies for the therapeutic suppression of neurodegenerative pathology

(2009) Anne Messer et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Prevention of oculopharyngeal muscular dystrophy by muscular expression of Llama single-chain intrabodies in vivo

(2009) Aymeric Chartier et al.   HUMAN MOLECULAR GENETICS

Detecting Morphologically Distinct Oligomeric Forms of α-Synuclein

(2009) Sharareh Emadi et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Phosphorylation of Threonine 3

(2009) Charity T. Aiken et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Intrabody Gene Therapy Ameliorates Motor, Cognitive, and Neuropathological Symptoms in Multiple Mouse Models of Huntington's Disease

(2009) A. L. Southwell et al.   JOURNAL OF NEUROSCIENCE

α-Synuclein and neuronal cell death

(2009) Mark R Cookson   Molecular Neurodegeneration

Serines 13 and 16 Are Critical Determinants of Full-Length Human Mutant Huntingtin Induced Disease Pathogenesis in HD Mice

(2009) Xiaofeng Gu et al.   NEURON

Intracellular targeting and clearance of oligomeric alpha-synuclein alleviates toxicity in mammalian cells

(2009) Bin Yuan et al.   NEUROSCIENCE LETTERS

Abberant α-Synuclein Confers Toxicity to Neurons in Part through Inhibition of Chaperone-Mediated Autophagy

(2009) Maria Xilouri et al.   PLoS One

Conformational Targeting of Fibrillar Polyglutamine Proteins in Live Cells Escalates Aggregation and Cytotoxicity

(2009) Erik Kvam et al.   PLoS One

Intrabody and Parkinson's disease

(2008) Chun Zhou et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Robustness promotes evolvability of thermotolerance in an RNA virus

(2008) Robert C McBride et al.   BMC EVOLUTIONARY BIOLOGY

Combinational approach of intrabody with enhanced Hsp70 expression addresses multiple pathologies in a fly model of Huntington’s disease

(2008) J. A. McLear et al.   FASEB JOURNAL

Huntington’s disease: degradation of mutant huntingtin by autophagy

(2008) Sovan Sarkar et al.   FEBS Journal

Single-chain intracellular antibodies inhibit influenza virus replication by disrupting interaction of proteins involved in viral replication and transcription

(2008) Muhammad Mahmood Mukhtar et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Suppression of neuropil aggregates and neurological symptoms by an intracellular antibody implicates the cytoplasmic toxicity of mutant huntingtin

(2008) Chuan-En Wang et al.   JOURNAL OF CELL BIOLOGY

Full-Length Human Mutant Huntingtin with a Stable Polyglutamine Repeat Can Elicit Progressive and Selective Neuropathogenesis in BACHD Mice

(2008) M. Gray et al.   JOURNAL OF NEUROSCIENCE

Intrabodies Binding the Proline-Rich Domains of Mutant Huntingtin Increase Its Turnover and Reduce Neurotoxicity

(2008) A. L. Southwell et al.   JOURNAL OF NEUROSCIENCE

The potential of intracellular antibodies for therapeutic targeting of protein-misfolding diseases

(2008) Alessio Cardinale et al.   TRENDS IN MOLECULAR MEDICINE

Camelid immunoglobulins and nanobody technology

(2008) S. Muyldermans et al.   VETERINARY IMMUNOLOGY AND IMMUNOPATHOLOGY

An scFv Intrabody against the Nonamyloid Component of α-Synuclein Reduces Intracellular Aggregation and Toxicity

(2007) Sandra M. Lynch et al.   JOURNAL OF MOLECULAR BIOLOGY