Peter Baas
Drexel University, PA·Neuroscience
Hereditary spastic paraplegia type 4 (SPG4-HSP) is an inherited nerve disease. It causes the legs to grow steadily weaker and stiffer, and many patients eventually need a wheelchair.
- Fields
Neuroscience
Pharmacology
Biomedical Engineering
- Topics
hereditary spastic paraplegia
spastin
microtubules
- Grants
- 1 known grant, through Mar 2028
What they work on
- Test whether lysosome-targeting intrabodies, antibody fragments made inside cells, effectively and sustainably deplete toxic mutant M1 spastin in hiPSC-derived motor cortical organoids without off-target toxicity.
- Determine whether degrading mutant M1 reverses disease features such as elevated HDAC6 activity, reduced microtubule acetylation and neurodegeneration in organoids from isogenic and SPG4-HSP patient-derived stem cell lines.
- Assess whether intrabodies also reduce normal wildtype M1 spastin and whether any resulting ill effects can be mitigated.
More about the research
The long nerve fibers that carry movement signals from the brain to the spinal cord, called corticospinal tracts, slowly die back. No therapies exist beyond easing symptoms. The disease is caused by mutations in the SPAST gene, which makes spastin, a protein that cuts microtubules (the internal scaffolding of cells). Recent studies point to one form of the protein, M1 spastin, as the main driver. When mutated, M1 becomes toxic and resists being broken down, so it builds up in neurons.
Why it matters
People with SPG4 hereditary spastic paraplegia have no treatment that slows their disease. If intrabodies can safely degrade toxic mutant spastin in patient-derived neurons, this approach could become a targeted therapy that halts and possibly reverses nerve degeneration, with potential for clinical translation.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
Eric M Morrow
Brown University, RI·Neuroscience
Map gene-expression and molecular pathway changes in embryonic brain of 17q12-deletion mice, testing whether reduced dosage of genes in the interval, including the Wnt-regulating transcription factor Lhx1, disrupts early brain development.
Gary Bassell
Emory University, GA·Neuroscience
Identify the RNA binding proteins and mRNA cargoes carried by the kinesin motor KIF1C, and test whether SPG58 pathogenic variants alter that binding, using mass spectrometry in CRISPR-generated KIF1C knockout human iPSC-derived motor neurons.
Anca Pasca
Stanford University, CA·Neuroscience
Identify when mitochondrial defects first appear during cortical development in 22q11.2 deletion syndrome, using advanced microscopy and functional assays in human cortical spheroids (hCS) and in forebrain sections from a mouse model.
The school
Drexel University: 11 labs on PI Pages, $170M a year on research, 282 research doctorates a year.
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