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Related Concept Videos

Gene Therapy00:59

Gene Therapy

22.1K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
22.1K

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Related Experiment Video

Updated: Apr 27, 2026

Transcriptome Analysis of Single Cells
07:27

Transcriptome Analysis of Single Cells

Published on: April 25, 2011

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Exploiting Single-Cell Tools in Gene and Cell Therapy.

Daniel Bode1,2, Alyssa H Cull3, Juan A Rubio-Lara3

  • 1Wellcome Medical Research Council (MRC) Cambridge Stem Cell Institute, University of Cambridge, Cambridge, United Kingdom.

Frontiers in Immunology
|July 29, 2021
PubMed
Summary
This summary is machine-generated.

Single-cell molecular tools offer unprecedented resolution of biological systems, identifying novel biomarkers and therapeutic targets. Advances in multi-omics and spatial technologies are paving the way for innovative cell and gene therapies.

Keywords:
CAR T cell therapycell therapydisease heterogeneitygene therapymultimodal omicsmultiomicsscRNA-seqsingle-cell sequencing

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Profiling Individual Human Embryonic Stem Cells by Quantitative RT-PCR
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A Combinatorial Single-cell Approach to Characterize the Molecular and Immunophenotypic Heterogeneity of Human Stem and Progenitor Populations
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A Combinatorial Single-cell Approach to Characterize the Molecular and Immunophenotypic Heterogeneity of Human Stem and Progenitor Populations

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Area of Science:

  • Biotechnology
  • Genomics
  • Molecular Biology

Background:

  • Single-cell molecular tools have advanced rapidly, enabling detailed analysis of individual cells.
  • These technologies provide high-resolution insights into cellular heterogeneity in fields like immunology and cancer biology.
  • Single-cell omics have identified biomarkers and targets previously undetectable by bulk methods.

Purpose of the Study:

  • To review emerging single-cell technologies and their integration for clinical applications.
  • To discuss the combination of single-cell molecular assays with functional data for mechanistic insights.
  • To explore the role of spatial transcriptomics and proteomics in cellular and gene therapy.

Main Methods:

  • Review of recent technological developments in single-cell sequencing and multi-omics.
  • Analysis of integrated single-cell data with functional and spatial information.
  • Discussion of CRISPR-based technologies, including base editors, for cell modulation.

Main Results:

  • Single-cell omics have identified novel disease biomarkers, cellular subsets, and therapeutic targets.
  • Integration of single-cell multi-omics with functional output and spatial location shows significant progress.
  • Emerging CRISPR technologies, like base editors, enhance prospects for cell and gene therapies.

Conclusions:

  • Single-cell technologies provide deep insights into complex biological systems and disease mechanisms.
  • Integration of multi-omics, functional, and spatial data is crucial for clinical translation.
  • Spatial transcriptomics and proteomics complement scRNA-seq, offering new avenues for cellular and gene therapy development.