Publications by authors named "P N Valdmanis"

We developed an hematopoietic stem cell (HSC) gene therapy approach that does not require cell transplantation. To achieve therapeutically relevant numbers of corrected cells, we constructed HSC-tropic HDAd5/35++ vectors expressing a 3' UTR truncated HMGA2 gene and a GFP reporter gene. A SB100x transposase vector mediated random integration of the tHMGA2/GFP transgene cassette.

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Article Synopsis
  • Proper transport of RNAs to synapses is really important for brain function and helps in learning and memory.
  • Alzheimer's disease causes damage in the brain that affects these RNA transports, leading to problems in brain communication.
  • Researchers found that in Alzheimer's patients, many RNA molecules are in the wrong places in the brain, and different types of circular RNAs may be linked to issues with a protein called tau, which could help in finding new treatments.
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  • MUC5AC and MUC5B are special proteins that help protect our bodies by catching germs and helping us clear mucus!
  • Researchers studied the differences in these proteins by looking at DNA from humans and primates and found that MUC5B is mostly the same in humans, while MUC5AC has many variations!
  • The study also showed that people from East Asia have unique versions of the MUC5AC protein that might have helped them in survival, while another version is more common in Europeans!
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The secreted mucins MUC5AC and MUC5B play critical defensive roles in airway pathogen entrapment and mucociliary clearance by encoding large glycoproteins with variable number tandem repeats (VNTRs). These polymorphic and degenerate protein coding VNTRs make the loci difficult to investigate with short reads. We characterize the structural diversity of and by long-read sequencing and assembly of 206 human and 20 nonhuman primate (NHP) haplotypes.

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Alzheimer's disease (AD), the most common aging-associated neurodegenerative dementia disorder, is defined by the presence of amyloid beta (Aβ) and tau aggregates in the brain. However, more than half of patients also exhibit aggregates of the protein TDP-43 as a secondary pathology. The presence of TDP-43 pathology in AD is associated with increased tau neuropathology and worsened clinical outcomes in AD patients.

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