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Neurodegeneration
Research Collection

Addgene has assembled the following to help you find information and plasmid collections that may help with your neurodegeneration research.

Neurodegenerative Disorders

Neurodegenerative diseases are a group of disorders characterized by the progressive degeneration and ultimate death of neurons. Common neurodegenerative diseases include Alzheimer’s, Parkinson’s, ALS, and Huntington’s Disease. These diseases persist for many years and lead to a major burden on the patients, their families and caretakers, and the health system in general. The required care for these millions of patients results in billions of dollars spent each year. Unfortunately, the causes of most neurodegenerative diseases are only partly understood, and there are no effective therapies to prevent, treat, or cure most of these disorders.

An integrated study of the overlapping biology of these diseases, rather than focusing on separate diseases, will help speed progress towards curing and treating them. Scientists are studying protein aggregation, learning about the blood brain barrier integrity, and using CRISPR screens to understand more globally how neurons function. They are also delving into new technologies and resources in order to gain more fundamental knowledge of how the brain works. The neurodegenerative disease field will advance more quickly with the avid sharing of data, tools, resources, methods, and results that are being adopted in the field.

Read on for a brief introduction to the genetics associated with some of the most common disorders followed by a list of some of the relevant collections available at Addgene.

Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS), sometimes referred to as Lou Gehrig’s disease, is a progressive disease affecting upper and lower motor neurons in the brain and spinal cord. Over time, a loss of motor neurons leads to limb weakness, difficulty speaking, swallowing, and breathing, and often results in death within 3–5 years from symptom onset. Disease mechanisms are still poorly understood, but several genes and mapped loci seem to be associated with the disease and implicate a wide range of cellular processes. The vast majority of cases (90–95%) of ALS are sporadic, having no prior family history. A small percentage (5–10%) are familial ALS cases having at least one other affected family member. These familial cases, usually inherited in an autosomal dominant manner, are associated with known genetic mutations in one of the following genes: SOD1, C9ORF72, TARDBP, FUS and OPTN.

Huntington's Disease

Huntington’s disease (HD) is a fatal autosomal dominant genetic disorder that causes the progressive degeneration of nerve cells in the brain. Symptoms include changes in behavior and emotion, uncontrolled movements, memory deficits, as well as difficulty speaking and swallowing. HD is caused by abnormal expansion of a repeating CAG triplet series within the huntingtin (HTT) gene. An individual with the mutated HTT gene will have huntingtin proteins with abnormally long polyglutamine sequences. These glutamine-rich sequences are prone to misfolding and aggregation and can interfere with protein-protein interactions and nerve cell function within the cell.

Parkinson's Disease

Parkinson’s disease (PD) is a chronic and progressive neurological disorder that primarily affects movement. More than ten million people worldwide are living with PD, mostly people over the age of 60. There is presently no cure or way to prevent progression of PD. Treatment consists mainly of managing symptoms through medication and surgery. PD primarily involves the malfunction and death of dopamine-producing neurons in the substantia nigra, one of the movement control centers in the brain. The cause of this neuron death is unknown. Despite the idiopathic (having no specific known cause) nature of the disease, a small portion of cases can be attributed to known genetic factors such as defects in LRRK2, SNCA, PARK7, or PINK1.

Alzheimer’s Disease

Alzheimer’s disease (AD) is the most common cause of dementia. Symptoms include memory loss, deficits in decision-making, and language abilities. The disease can be classified as early-onset, where symptoms appear between a person’s thirties and mid-sixties, or late-onset, where symptoms appear during or after a person's mid-sixties. The early-onset form accounts for less than 10 percent of all cases of Alzheimer's disease. Of these early-onset forms, a fraction of these are due to a mutation in one of three different inherited genes: Presenilin 1, Presenilin 2, and APP. The majority (> 90%) of individuals develop late-onset AD. There is no clear association between a gene and late-onset AD. It is more likely a combination of gene variation and lifestyle or environmental factors. For example, variations of Apolipoprotein E (APOE), such as the ε4 allele, are a risk factor for late-onset AD. Many more genes have been associated with Alzheimer's disease, through large genome-wide association studies (GWAS) or other large scale studies. Researchers are now investigating the role that these additional genes may play in Alzheimer's disease.

Research Tools

Neurodegeneration Plasmid Collection

A collection of plasmids expressing wild type or mutant ORFs, gene fragments, and gRNAs for genes linked to neurodegenerative disorders. Find plasmids expressing HTT, C9ORF72, LRRK2, Synuclein, and more.

  • Study ER structure and function with siRNA-resistance SigmaR1 constructs. (Sawyer et al. Dev Cell. 2024)
  • Image exocytosis with VAMP2/3 plasmids to study CNS myelination. (Lam et al. Nat Commun. 2022)
  • Endogenously tag gene variants for Alzheimer's disease and related dementias with plasmids from the iPSC Neurodegenerative Disease Initiative Collection.
  • Use the 3xFlag-eGFP-Flag-SETX construct for subcellular localization studies with GFP, or IP with Flag tag. (Bennett et al. Heliyon. 2020)
  • Use VAP expression plasmids to study ER contact with other organelles. (Mattia et al. EMBO Rep. 2018)
  • Study intrinsically disordered protein regions with FUS and HNRNPA1 plasmids. (Shin et al. Cell. 2017)
  • Study amyloid deposition with APOE2 constructs. (Hudry et al. Sci Transl Med. 2013)
  • Express human DNMT1 with a Myc tag. (Li et al. J Biol Chem. 2006)
  • Express human ATM with Flag and His tags. (Canman et al. Science. 1998)
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CRISPR Tools

Find CRISPR pooled libraries available from Addgene, sortable by type of genetic modification and target species. You can also find our guide to using plasmid pooled libraries.

  • Use the ultracompact and programmable endonuclease, TnpBmax, for genome editing. (Marquart et al. Nat Methods. 2024)
  • Base edit human mitochondrial DNA using mitoBEs. (Yi et al. Nat Biotechnol. 2023)
  • Initiate heritable gene silencing with CRISPRoff-v2.1. (Nuñez et al. Cell. 2021)
  • Conduct multimodal genetic screens in human iPSC-derived neurons with a CRISPRi-based platform. (Tian et al. Neuron. 2019)
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AAV Viral Preps

Find AAV viral preps for systemic delivery of viral particles that will target the central nervous system, peripheral nervous system, vesicular brain cells and more.

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iPSC Differentiation Factors

Find plasmids used to transform iPSCs and fibroblasts into neurons and more.

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Antibodies

Find information on Addgene's collection of plasmid-based antibodies, including monoclonal antibodies, scFvs, and nanobodies.

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Fluorescent Proteins

Find information on Addgene's collections of fluorescent proteins, including Markers of Subcellular Localization, Optogenetics Plasmids, and Chemogenetics Plasmids.

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Other Resources

BRAIN Initiative Collection
The BRAIN Initiative supports the development of a diverse portfolio of biomolecular tools and emphasizes their rapid and broad dissemination to the research community. This collection highlights plasmids created with support from the BRAIN Initiative.
Jackson Laboratory (Link opens in a new window)
A collection of neurological mouse models including complex and monogenic diseases.
Alzforum (Link opens in a new window)
A news website and information resource dedicated to helping researchers accelerate discovery and advance development of diagnostics and treatments for Alzheimer’s disease and related disorders. The site curates information on useful resources such as:
Michael J Fox Foundation (Link opens in a new window)
A foundation dedicated to finding a cure for Parkinson's disease through an aggressively funded research agenda and to ensuring the development of improved therapies for those living with Parkinson's today.
CHDI Foundation (Link opens in a new window)
An organization with a mission to rapidly develop therapeutics that will slow the progression of Huntington’s disease. The foundation offers curated information on tools and reagents (Link opens in a new window) such as huntingtin cDNAs, antibodies, and cell lines.

This resource was created with support from the Chan Zuckerberg Initiative (Link opens in a new window).