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Serge Przedborski

Columbia University, NY·Neuroscience

Parkinson's disease (PD) and related disorders are called synucleinopathies. After Alzheimer's disease, they are the most common group of brain-degenerating diseases.

Fields

Neuroscience

Pharmacology

Medicine & Clinical Research

Topics

Parkinson's disease

synucleinopathy

alpha-synuclein

Grants
1 known grant, through May 2028

What they work on

  1. Evaluate whether virus-delivered anti-α-Syn single-chain antibody fragments (scFvs) improve gut function and reduce gut pathology in transgenic L61 mice, which overproduce alpha-synuclein, using GI function tests and tissue analysis.
  2. Assess whether gut-targeted scFv gene therapy protects movement, cognition and brain tissue in mice given gut injections of α-Syn preformed fibrils (PFF), seed-like clumps that spread pathology toward the brain.
  3. Compare secreted and non-secreted anti-α-Syn scFvs delivered by AAV-MaCPNS2, a viral vector that reaches the gut's nervous system, including blood toxicology and organ tissue checks for safety.

More about the research

In these diseases, clumps of a small nerve-ending protein called alpha-synuclein (α-Syn) build up in nerve cells. PD is diagnosed by its movement problems, but other symptoms often appear years earlier. More than 80% of patients report constipation, which can begin decades before diagnosis. About half of patients have α-Syn clumps in the gut before the brain. This suggests the disease may start in the gut's own nervous system and spread to the brain.

Why it matters

Gut problems such as constipation affect most people with Parkinson's disease and can appear long before movement symptoms. If antibody fragments delivered to the gut can clear abnormal alpha-synuclein, this approach could lead to an early-stage treatment for Parkinson's disease and related dementing conditions. It could also help test the idea that the disease spreads from the gut to the brain.

Known grants

From NIH and NSF research grants. Labs often have other funding we can't see.

Gene Therapy for Non-Motor Defects in Synucleinopathy

NIH · Jun 2026 – May 2028

Closest labs

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Test whether the glycopeptide PNA5 improves memory and thinking deficits in male mice injected with alpha-synuclein preformed fibrils, compared with scrambled-glycopeptide and vehicle controls, using behavioral tests.

Shermali Gunawardena

University at Buffalo, NY·Neuroscience

Test whether the N-terminal region of α-synuclein, the Parkinson's-linked protein, controls how mitochondria break into smaller pieces, using transgenic Drosophila carrying N-terminal deletions.

Qinglan Ling

University of Massachusetts Chan Medical School, MA·Genetics·Early-career

Develop and optimize a mitochondria-targeting base editor, a tool that changes single DNA letters, to create a truncating mutation in the mouse MTATP6 gene and generate a conditional mouse model.

The school

Columbia University: 89 labs on PI Pages, $1.4B a year on research, 625 research doctorates a year.

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