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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:

  • Eif2b3 mutants recapitulate phenotypes of vanishing white matter disease and validate novel disease alleles in zebrafish. Lee et al. Hum Mol Genet. 2021 Apr 27.
  • New lentiviral vector expressing human DNAJC6 to study Parkinson's Disease. Wulansari et al. Sci Adv. 2021 Feb 17.
  • Lentiviral constructs of ALS-associated mutations in ANXA11 show their role in RNA granule transport. Liao et al. Cell. 2019 Sep 19.
  • Plasmids from the iPSC Neurodegenerative Disease Initiative (iNDI) Collection allow endogenous tagging of gene variants for Alzheimer's disease and related dementias (ADRD) research.

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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:

  • CAKE: A CRISPR/Cas9-based tool for multiplex genome editing in neurons. Droogers et al. bioRxiv. 2022.
  • CRISPRoff allows for programmable and maintainable DNA methylation and gene repression. Nunez et al. Cell. 2021 Apr 7.

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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:

  • The new BiPOLES (Bidirectional Pair of Opsins for Light-induced Excitation and Silencing) vectors have two-opsins in one protein, so you can excite and inhibit the same neuron. Vierock et al. Nat Commun. 2021 Jul 26.
  • Recently expanded selection of GCaMP8 preps from the Genie Project in ready to use AAV viral preps.

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iPSC Differentiation Factors

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

New and Noteworthy:

  • Efficient generation of dopaminergic induced neuronal cells with midbrain characteristics. Ng et al. Stem Cell Reports. 2021 Jul 13.
  • Species-wide method for deriving iPSCs that have a neural stem cell-like state during the reprogramming. Yoshimatsu et al. Stem Cell Reports. 2021 Mar 23.

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Antibodies

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

New and Noteworthy:

  • Addgene now distributes ready-to-use recombinant antibodies made from plasmids in our collection! These monoclonal antibodies are produced in-house and undergo application-specific validation and quality control by Addgene as well as by our trusted partner labs.

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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.
  • 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.