journal cover

Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model

Neuron

Dec 14, 2019

Lien NguyenLaura P W Ranum

Abstract

The intronic C9orf72 G4C2 expansion, the most common genetic cause of ALS and FTD, produces sense- and antisense-expansion RNAs and six dipeptide repeat-associated, non-ATG (RAN) proteins, but their roles in disease are unclear. We generated high-affinity human antibodies targeting GA o...read more

Mentioned in this Paper

C9orf72 protein, human
Bacitracin
Blood - Brain Barrier Anatomy
RNA, Untranslated
Antibody Therapy
Nerve Degeneration
Ran GTPase Binding
TRIM21 protein, human
Amyotrophic Lateral Sclerosis
Cell Growth
19
2
Paper Details
References
  • References
  • Citations
  • finger pointing at paper

    References currently unavailable

    We're still populating references for this paper, please check back later.
  • References
  • Citations
  • quote and clock

    No citations available

    This paper may not have been cited yet.

Similar Papers Found In These Feeds

Autophagy & Model Organisms

Autophagy is a cellular process that allows degradation by the lysosome of cytoplasmic components such as proteins or organelles. Here is the latest research on autophagy & model organisms

Proteostasis

Proteostasis enables the maintenance of protein homeostasis via modulation of protein translation, enhancement of chaperone capacity and the prompt clearance of misfolded proteins. It is affected in several neurodegenerative diseases. Here is the latest research.

Misfolding & Aggregation Diseases

Misfolding and aggregation of proteins can lead to several diseases. For instance, misfolding of prion or tau proteins are associated with several neurodegenerative diseases. Here is the latest research on diseases caused by protein misfolding and aggregation.

ALS - Pathogenic Mechanisms

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder characterized by muscle weakness. Here is the latest research investigating pathogenic mechanisms that underlie this genetically heterogeneous disorder.

Neuroscience Journals

Discover the latest neuroscienceresearch in this collection of the top neuroscience journals.

ALS

Amyotrophic Lateral Sclerosis (ALS), also known as motor neuron disease, is associated with the death of neurons that control voluntary muscles. Discover the latest research on ALS here.

RNA & Axonal Functions

This feed focuses on subcellular and translation regulation of RNA in Axons and how this influences Axonal Functions. Discover the latest research here.

Neuromuscular Disorders

Neuromuscular disorders result in impaired functioning of the muscles. Discover the latest research on these disorders, including ALS, and the underlying genetic aspects here.

Blood Brain Barrier Chips

The blood brain barrier (BBB) is comprised of endothelial cells that regulate the influx and outflux of plasma concentrations. Lab-on-a-chip devices allow scientists to model diseases and mechanisms such as the passage of therapeutic antibodies across the BBB. Discover the latest research on BBB chips here.

Frontotemporal Lobar Degeneration

Frontotemporal lobar degeneration or frontotemporal dementia refers to a group of uncommon disorders that occur as a result of progressive nerve cell loss in the frontal and temporal lobes of the brain. Here is the latest research.

Neurodegeneration: Autophagy

Given that progression of neurodegenerative diseases can be driven by aggregation of misfolded proteins, autophagic activity is through to modulate the severity of neurodegenerative diseases. Here is the latest research on the influence of autophagy on neurodegeneration.

RNA & Tau in Frontotemporal Dementia

Tau mutations in frontotemporal dementia and parkinsonism linked to chromosome 17 (ftdp-17) are associated with changes in alternative RNA splicing. Here is the latest research on RNA & Tau in Frontotemporal Dementia.

Autophagy & Disease

Autophagy is an important cellular process for normal physiology and both elevated and decreased levels of autophagy are associated with disease. Here is the latest research.

ALS - Phenotypes

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder characterized by muscle weakness. Here is the latest research investigating phenotypes associated with this genetically heterogeneous disorder.

Brain developing: Influences & Outcomes

This feed focuses on influences that affect the developing brain including genetics, fetal development, prenatal care, and gene-environment interactions. Here is the latest research in this field.

Cell Adhesion Molecules in the Brain

Cell adhesion molecules found on cell surface help cells bind with other cells or the extracellular matrix to maintain structure and function. Here is the latest research on their role in the brain.

ALS & FTD: TDP-43

ALS shares with a considerable proportion of FTD cases the same neuropathological substrate, namely, inclusions of abnormally phosphorylated protein tdp-43 (ptdp-43). Here are the latest discoveries pertaining to ptdp-43 and these diseases.

Frontotemporal Dementia

Frontotemporal dementia (FTD) refers to disorders caused by progressive neuronal loss in the frontal and temporal lobes of the brain. Here is the latest research on FTD and FTD-associated disorders.

Lysosome & C9orf72

This feed focuses on the C9orf72 protein and its possible role in lysosome function and implication in frontotemporal dementia and amyotrophic lateral sclerosis

Motor Neuron Disease

Motor neuron diseases such as amyotrophic lateral sclerosis and spinal muscular atrophy are progressive neurodegenerative diseases that result in the death of motor neurons. Discover the latest research on motor neuron disease here.

Blood Brain Barrier

The blood brain barrier is a border that separates blood from cerebrospinal fluid. Discover the latest search on this highly selective semipermeable membrane here.

ALS: Therapies

Amyotrophic Lateral Sclerosis (ALS), also known as motor neuron disease, is associated with the death of neurons that control voluntary muscles. Discover the latest research on ALS therapies here.

CZI Neurodegeneration Challenge Network

The Neurodegeneration Challenge Network aims to provide funding for and to bring together researchers studying neurodegenerative diseases. Find the latest research from the NDCN grantees here.

© 2020 Meta ULC. All rights reserved

Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model

Neuron

Dec 14, 2019

Lien NguyenLaura P W Ranum

PMID: 31831332

DOI: 10.1016/j.neuron.2019.11.007

Abstract

The intronic C9orf72 G4C2 expansion, the most common genetic cause of ALS and FTD, produces sense- and antisense-expansion RNAs and six dipeptide repeat-associated, non-ATG (RAN) proteins, but their roles in disease are unclear. We generated high-affinity human antibodies targeting GA o...read more

Mentioned in this Paper

C9orf72 protein, human
Bacitracin
Blood - Brain Barrier Anatomy
RNA, Untranslated
Antibody Therapy
Nerve Degeneration
Ran GTPase Binding
TRIM21 protein, human
Amyotrophic Lateral Sclerosis
Cell Growth
19
2

Similar Papers Found In These Feeds

Autophagy & Model Organisms

Autophagy is a cellular process that allows degradation by the lysosome of cytoplasmic components such as proteins or organelles. Here is the latest research on autophagy & model organisms

Proteostasis

Proteostasis enables the maintenance of protein homeostasis via modulation of protein translation, enhancement of chaperone capacity and the prompt clearance of misfolded proteins. It is affected in several neurodegenerative diseases. Here is the latest research.

Related Papers

Paper Details
References
  • References
  • Citations
  • finger pointing at paper

    References currently unavailable

    We're still populating references for this paper, please check back later.
  • References
  • Citations
  • quote and clock

    No citations available

    This paper may not have been cited yet.
/papers/antibody-therapy-targeting-ran-proteins-rescues/31831332