Publications by authors named "L De la Barrera"

(1) Background: Children with medical complexity (CMC) and neurologic impairment (NI) are a growing population in pediatric intensive care units (PICUs). (2) Objective: Our aim was to explore and describe the experiences and beliefs of PICU providers caring for CMC with NI. (3) Methods: A qualitative interview-based study was conducted.

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Stargardt disease is a currently untreatable, inherited neurodegenerative disease that leads to macular degeneration and blindness due to loss-of-function mutations in the ABCA4 gene. We have designed a dual adeno-associated viral vector encoding a split-intein adenine base editor to correct the most common mutation in ABCA4 (c.5882G>A, p.

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Objective: To estimate the direct costs associated with the diagnosis, treatment, and control of measles cases in Brazil from 2018 to 2020.

Methods: This cost-of-illness study utilized a prevalence-based approach, considering direct costs incurred by the Brazilian Public Health System (SUS) related to measles outbreaks, including costs of inpatient care, outpatient care, and laboratory tests, as well as measles-containing vaccines and laboratory tests (viral isolation) used for outbreak control. Costs are presented in 2020 US dollars.

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Article Synopsis
  • The study aimed to understand parents' views on using home monitoring technologies for early childhood research, focusing on their concerns and enthusiasm.
  • Participants included new and expecting parents who took part in interviews about hypothetical studies involving wearable devices, audio monitoring, and environmental sampling.
  • Themes from the interviews indicated that while parents were generally positive about participation, they raised concerns about safety, data privacy, and the logistical challenges of involvement, highlighting the need for trust and transparency from researchers.
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Article Synopsis
  • * The huR83C mouse model replicates the disease phenotype and has been used to test the effectiveness of BEAM-301, a treatment that utilizes lipid nanoparticles and adenine base editing to correct the harmful G6PC1-R83C variant.
  • * BEAM-301 has shown the ability to correct about 60% of the variant in liver cells, restore blood sugar control, improve overall health, and increase survival rates in mice, indicating its potential as a therapeutic option for patients with this specific genetic mutation
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