Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.

Lin Y, Liou B, Fannin V, Adler S, Mayhew CN, Hammonds JE, Hu YC, Tchieu J, Zhang W, Zhao X, Beres RL, Setchell KDR, Kaynak A, Qi X, Feldman RA, Sun Y

Open source

DOI
10.7554/elife.109518
Published
2026 Jun 23
Container
eLife
Publisher
Not recorded
Open access
yes

Credibility signals

limited evidence Score 45/100 under policy 1.0.0. This is a metadata assessment, not a judgment of the paper's conclusions.

Show all credibility signals

Cite this work

BibTeX

@article{allodium:10.7554/elife.109518,
  title = {Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.},
  author = {Lin Y and Liou B and Fannin V and Adler S and Mayhew CN and Hammonds JE and Hu YC and Tchieu J and Zhang W and Zhao X and Beres RL and Setchell KDR and Kaynak A and Qi X and Feldman RA and Sun Y},
  year = {2026},
  journal = {eLife},
  doi = {10.7554/elife.109518},
  url = {https://doi.org/10.7554/elife.109518}
}

RIS

TY  - JOUR
TI  - Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.
AU  - Lin Y
AU  - Liou B
AU  - Fannin V
AU  - Adler S
AU  - Mayhew CN
AU  - Hammonds JE
AU  - Hu YC
AU  - Tchieu J
AU  - Zhang W
AU  - Zhao X
AU  - Beres RL
AU  - Setchell KDR
AU  - Kaynak A
AU  - Qi X
AU  - Feldman RA
AU  - Sun Y
PY  - 2026
JO  - eLife
DO  - 10.7554/elife.109518
UR  - https://doi.org/10.7554/elife.109518
ER  - 

APA

Y, L., B, L., V, F., S, A., CN, M., JE, H., YC, H., J, T., W, Z., X, Z., RL, B., KDR, S., A, K., X, Q., RA, F., & Y, S. (2026). Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.. eLife. https://doi.org/10.7554/elife.109518

Source records