Miguel C. Santoscoy

ORCID: 0000-0003-0249-4529
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About
Contact & Profiles
Research Areas
  • RNA Interference and Gene Delivery
  • Virus-based gene therapy research
  • Bacterial Genetics and Biotechnology
  • Microbial Metabolic Engineering and Bioproduction
  • Enzyme Catalysis and Immobilization
  • Bacteriophages and microbial interactions
  • Neuroscience and Neural Engineering
  • RNA and protein synthesis mechanisms
  • Biofuel production and bioconversion
  • Neuroinflammation and Neurodegeneration Mechanisms
  • interferon and immune responses
  • Neurogenetic and Muscular Disorders Research
  • CRISPR and Genetic Engineering
  • Neurological disorders and treatments
  • Advanced Chemical Sensor Technologies
  • Pluripotent Stem Cells Research
  • Biosensors and Analytical Detection
  • Advanced biosensing and bioanalysis techniques
  • Bacterial biofilms and quorum sensing
  • Extracellular vesicles in disease
  • Microbial Fuel Cells and Bioremediation
  • Immune Response and Inflammation
  • Thermochemical Biomass Conversion Processes
  • S100 Proteins and Annexins

Massachusetts General Hospital
2023-2024

Harvard University
2023-2024

Iowa State University
2017-2022

Bissell (United States)
2018

Systemic administration of adeno-associated virus (AAV) vectors for spinal cord gene therapy has challenges including toxicity at high doses and pre-existing immunity that reduces efficacy. Intrathecal (IT) delivery AAV into cerebral fluid can avoid many issues, although distribution the vector throughout is limited, entry to periphery sometimes initiates hepatotoxicity. Here we performed biopanning in non-human primates (NHPs) with an IT injected AAV9 peptide display library. We identified...

10.1016/j.ymthe.2024.05.040 article EN cc-by-nc-nd Molecular Therapy 2024-06-05

Adeno-associated virus (AAV) vectors are currently the most efficient option for intracranial gene therapies to treat neurodegenerative disease. Increased efficacy and safety will depend upon robust specific expression of therapeutic genes into target cell-types within human brain. In this study, we set out with two objectives: (1) identify capsids broader transduction striatum injection in mice (2) test a truncated choline acetyltransferase (ChAT) promoter that would allow selective...

10.1016/j.omtm.2023.05.001 article EN cc-by-nc-nd Molecular Therapy — Methods & Clinical Development 2023-05-09

10.1016/j.ymben.2021.03.012 article EN publisher-specific-oa Metabolic Engineering 2021-04-02

Outer membrane protein A (OmpA) is one of the most abundant outer proteins Gram-negative bacteria and known to have patterns sequence variations at certain amino acids—allelic variation—in Escherichia coli . Here we subjected seven exemplar OmpA alleles expressed in a K-12 (MG1655) Δ ompA background further characterization. These were observed significantly impact cell surface charge (zeta potential), hydrophobicity, biofilm formation, sensitivity killing by neutrophil elastase, specific...

10.1371/journal.pone.0276046 article EN cc-by PLoS ONE 2022-10-13

Abstract Systemic administration of adeno-associated virus (AAV) vectors for spinal cord gene therapy has challenges including toxicity at high doses and pre-existing immunity that reduces efficacy. Intrathecal delivery AAV into the cerebral fluid (CSF) can avoid many issues systemic delivery, although achieving broad distribution vector transgene expression throughout is challenging entry to periphery occurs, sometimes initiating hepatotoxicity. Here we performed two rounds in vivo...

10.1101/2023.09.13.557506 preprint EN cc-by-nc-nd bioRxiv (Cold Spring Harbor Laboratory) 2023-09-13

Adeno-associated viruses (AAV) are promising vectors for gene therapy due to their efficacy in vivo. However, there is room improvement address key limitations such as the pre-existing immunity AAV patients, high-dose toxicity, and relatively low efficiency some cell types. This study introduces a metabolic engineering approach, using knockout of enzyme phosphatidylserine synthase 1 (PTDSS1) increase abundance extracellular vesicle-enclosed (EV-AAV) relative free supernatant producer cells,...

10.1101/2024.08.16.608303 preprint EN bioRxiv (Cold Spring Harbor Laboratory) 2024-08-19
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