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Welcome to Addgene's Neurodegeneration Research Landing Page!
Here you’ll 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 gene.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

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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.

New and Noteworthy:

  • CRISPR prime editing constructs targeting KCNA1. Liu et al. Cell Discov. 2020 Apr 28.
  • TBP repeat expansion constructs. Basu et al. Cell. 2020 May 28.
  • hnRNP2 RRM constructs used in structural and aggregation studies. Ryan et al. Nucleic Acids Res. 2020 Sep 1.

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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.

New and noteworthy:

  • CROPseq and LENTIGuide constructs used in two-step guide-barcode cloning and optical pooled screens. Feldman et al. Cell. 2019 Oct 17.
  • dCas9-KRAB constructs used in CRISPRi in iPSC derived neurons. Tian et al. Neuron. 2019 Aug 5.
    • CRISPRbrain Data Commons for functional genomics screens in differentiated human cell types. Search data from existing screens or contribute your own results.
  • CRISPRi and CRISPRa constructs for AAV delivery in neurons. Lau et al. Mol Ther Nucleic Acids. 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.

New and noteworthy:

  • A decision tree for helping you choose the best serotype for systemic delivery.
  • PHP.V1 serotype for efficient transduction of vesicular brain cells via systemic delivery in adult animals.
  • University of Florida Serotype Testing Panel for the Eye and Brain.

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iPSC Differentiation Factors

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

New and Noteworthy:


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Antibodies

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

New and Noteworthy:


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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.

New and Noteworthy:


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.
  • Meta Over 220 different feeds to keep you on top of current Neurodegeneration research.
  • Jackson Laboratory collection of neurological mouse models including complex and monogenic diseases.
  • Alzforum is 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 is 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.
    • The Michael J. Fox Foundation has made a number of tools available to the scientific community at low cost, with rapid delivery. Includes antibodies, viral vectors, animal models, purified protein, and more.
    • Plasmids provided by the foundation can be found on our Michael J. Fox Foundation Plasmid Resource page.
  • CHDI Foundation is 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 such as huntingtin cDNAs, antibodies, and cell lines.
  • Neurodegeneration Method Development Community at Protocols.io.

This resource is supported by the Chan Zuckerberg Initiative.