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Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
Published on: December 10, 2021
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Germline transmission in transgenic Huntington's disease monkeys
Sean Moran1, Tim Chi1, Melinda S Prucha1
1Department of Human Genetics, Emory University School of Medicine, Atlanta, Georgia, USA; Division of Neuropharmacology and Neurologic Diseases, Yerkes National Primate Research Center, Emory University, Atlanta, Georgia, USA.
Theriogenology
|April 29, 2015
Summary
Researchers confirmed stable germline transmission of the mutant huntingtin (mHTT) transgene in Huntington
Area of Science:
- Neuroscience
- Genetics
- Primate Models
Background:
- Transgenic nonhuman primates offer valuable models for neurologic and neurodegenerative diseases due to human-like brain anatomy and function.
- Huntington's disease (HD) monkeys exhibit clinical features mirroring human HD patients, making them suitable for preclinical research.
- Limited colony expansion currently restricts the widespread use of HD monkeys in preclinical studies.
Purpose of the Study:
- To confirm germline transmission of the mutant huntingtin (mHTT) transgene in HD monkeys.
- To establish methods for expanding HD monkey colonies for future research.
Main Methods:
- Generated embryonic stem cells from three male HD monkey founders (F0).
- Produced second-generation offspring (F1) using artificial insemination (intrauterine insemination) with semen from F0 HD monkeys.
- Confirmed mHTT transgene expression in F1 HD monkeys and derived induced pluripotent stem cells using quantitative real-time polymerase chain reaction.
Main Results:
- Confirmed germline transmission of the mHTT transgene in F0 embryonic stem cells and F1 offspring.
- Produced five F1 offspring from 15 inseminated females (33% success rate).
- Achieved 100% germline transmission rate in F1 offspring from one specific HD founder (rHD8), resulting in two transgenic HD monkeys.
- Verified mHTT expression in F1 HD monkeys and their induced pluripotent stem cells.
Conclusions:
- Demonstrated stable germline transmission and expression of the mHTT transgene in HD monkeys.
- This breakthrough suggests a viable strategy for expanding HD monkey colonies.
- Facilitates future preclinical and biomedical research for Huntington's disease.
Keywords:
Germline transmissionSecond generationTransgenic Huntington's disease monkeyUltrasound-guided intrauterine insemination
