Contemporary and emerging technologies for research in children's rare and interstitial lung disease.

Pediatr Pulmonol

Department of Pediatrics, Pulmonology Division, Program for Rare and Interstitial Lung Disease, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.

Published: September 2024

AI Article Synopsis

  • Recent advancements in genetics have identified many children's interstitial and rare lung disease (chILD) disorders, but effective treatments and a complete understanding of their causes are still missing.
  • New technologies like high-throughput sequencing and spatial analysis are revolutionizing the study of cellular biology, allowing researchers to analyze thousands of genes and their roles in disease and normal function.
  • Innovative methods such as gene editing and organoid creation are being combined with data science to enhance research efforts, ultimately aiming to improve our understanding and treatment of chILD disorders.

Article Abstract

Although recent decades have seen the identification, classification and discovery of the genetic basis of many children's interstitial and rare lung disease (chILD) disorders, detailed understanding of pathogenesis and specific therapies are still lacking for most of them. Fortunately, a revolution of technological advancements has created new opportunities to address these critical knowledge gaps. High-throughput sequencing has facilitated analysis of transcription of thousands of genes in thousands of single cells, creating tremendous breakthroughs in understanding normal and diseased cellular biology. Spatial techniques allow analysis of transcriptomes and proteomes at the subcellular level in the context of tissue architecture, in many cases even in formalin-fixed, paraffin-embedded specimens. Gene editing techniques allow creation of "humanized" animal models in a shorter time frame, for improved knowledge and preclinical therapeutic testing. Regenerative medicine approaches and bioengineering advancements facilitate the creation of patient-derived induced pluripotent stem cells and their differentiation into tissue-specific cell types which can be studied in multicellular "organoids" or "organ-on-a-chip" approaches. These technologies, singly and in combination, are already being applied to gain new biological insights into chILD disorders. The time is ripe to systematically apply these technologies to chILD, together with sophisticated data science approaches, to improve both biological understanding and disease-specific therapy.

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Source
http://dx.doi.org/10.1002/ppul.26490DOI Listing

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