Compact Calm1 promoter enables AAV mediated neuron-targeted expression in human iPSC-derived brain organoids

Ina Klockner, Meghana Yeturi, Trinity E Rust, Jason L Stein, Mark J Zylka · 2025

Read Compact Calm1 promoter enables AAV mediated neuron-targeted expression in human iPSC-derived brain organoids by Ina Klockner, Meghana Yeturi, Trinity E Rust, Jason L Stein, Mark J Zylka (2025) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-12738672.txt.

Description

Ina Klockner and co-authors test a compact gene switch in a 2025 study using human brain organoids. Gene therapy viruses have tight space limits, so small switches that turn genes on only in neurons are prized. A new switch from a calm related gene is far shorter than the standard one and worked well in animals.

The team placed each switch in a virus carrying a marker gene and grew the viruses in lab grown brain tissue from human stem cells. Both switches drove marker activity in neurons at much the same level. Tissue staining backed the match. The finding supports the small switch as a space saving choice for future brain gene therapies.

Catalog record

CREATORIna Klockner, Meghana Yeturi, Trinity E Rust, Jason L Stein, Mark J Zylka
YEAR2025
COLLECTIONPAPERS
FORMATTXT
FILE SIZE23 KB
VERSION1.0
LICENSECC BY 4.0
RIGHTSCC BY
SOURCEEurope PMC

Source: https://europepmc.org/articles/PMC12738672

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