Biodistribution Maximize gene therapy performance

Single-cell sequencing can be used in biodistribution studies to optimize the safety and efficacy of gene editing by validating transgene expression in cells, revealing cell tropism, and studying transcriptomic response after administration.

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Apply single-cell sequencing in biodistribution studies

Biodistribution at single-cell resolution is a powerful tool for the design, selection, and optimization of adenoviral, lentiviral vectors, or other nanoparticle characteristics such as transduction efficacy, cell tropism, and post-administration side effects.

Our R&D team is collaborating with biopharmaceutical companies to develop transgene detection at single-cell resolution, enabling unprecedented single-cell readouts for gene therapy efficacy and safety.

Want to bring the benefits of single-cell sequencing to your biodistribution study? Speak to our R&D representatives to design a bespoke single-cell analysis customized to your specific biodistribution study goals.

Application note: how single-cell sequencing can optimize gene therapy biodistribution studies

Read our Biodistribution Application Note

Learn about our new approach to single-cell biodistribution: a transgene expression assay for AAV drug developers.

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Detect transgene expression to select optimal viral vectors

Single-cell sequencing in biodistribution studies can validate and quantify transduction efficacy per cell and per tissue to rank candidate vectors.

For lowly expressed transgenes, our R&D scientists can explore amplification maximization steps to fish out your transgene sequence.

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Understand tropism to cellular subtypes

Biodistribution studies have been traditionally limited to bulk RNA sequencing on isolated tissue biopsies to measure local transduction rates. However single-cell sequencing characterizes biodistribution to different cellular subtypes, enabling candidate vector stratification and optimization of transduction efficiency and specificity.

Assess safety by studying post-administration gene expression differences

Coupling single-cell transgene detection with single-cell RNA sequencing reveals how therapeutic transgene expression directly influences the cellular transcriptome. The combined readouts informs on vector specificity and efficiency as well as therapeutic efficacy and safety.

Single Cell Discoveries oncology single-cell sequencing

Case studies
April 7, 2021

Snake Venom Gland Organoids

Three PhD students, under the supervision of Prof. Dr Hans Clevers in molecular genetics and snake expert Prof. Dr Freek Vonk, conducted this remarkable research in which they developed snake venom-producing organoids. The organoids were characterized using SORT-seq. Here, we explain how the project was done.

Developmental Biology
SORT-seq
August 16, 2021

Colorectal Zebrafish Xenograft Model

This research, conducted at the Champalimaud Foundation in Lisbon, is focused on the immune response after implantation of human cancer cells in zebrafish. The cells in the implanted tumors were characterized using SORT-seq. Here, we explain how SORT-seq contributed to this remarkable study.

Immunology
Oncology
SORT-seq
November 4, 2022

Single-Cell Atlas of the Human Cornea

Read about the eye-opening collaboration of Single Cell Discoveries and MERLN Institute for Technology-Inspired Regenerative Medicine.

10x
Regenerative Medicine

Recent publications

How can we help?

Want to supercharge your biodistribution study with single-cell insights?

Connect with our PhD-level scientists to discuss your biological question, timeline, sample types, and other customizations for your single-cell analysis.