CRISPR/Cas9-generated mouse model with humanizing single-base substitution in the Gnao1 for safety studies of RNA therapeutics

AV Polikarpova, TV Egorova, EA Lunev… - Frontiers in Genome …, 2023 - frontiersin.org
The development of personalized medicine for genetic diseases requires preclinical testing
in the appropriate animal models. GNAO1 encephalopathy is a severe neurodevelopmental …

New personalized genetic mouse model of Lesch-Nyhan syndrome for pharmacology and gene therapy

SY Yu - Research Results in Pharmacology, 2018 - cyberleninka.ru
Introduction: Lesch-Nyhan syndrome is a clinical and laboratory disorder caused by X-linked
disruption of the purine metabolism. The deletion in the HPRT1 gene leads to the …

Использование технологии CRISPR/Cas9 при создании животных-продуцентов рекомбинантных белков человека в проектах НОЦ мирового уровня « …

АВ Дейкин, ВО Солдатов, МВ Корокин… - Достижения науки и …, 2020 - cyberleninka.ru
Создание трансгенных организмов открывает ряд направлений их биомедицинского
использования, к числу которых относится производство рекомбинантных белков …

[PDF][PDF] New personalized genetic mouse model of Lesch-Nyhan syndrome for pharmacology and gene therapy. Research Results in Pharmacology 4 (4): 115–122

VA Kalmykov, PA Kusov, MI Yablonskaia… - 2018 - pdfs.semanticscholar.org
Abstract Introduction: Lesch-Nyhan syndrome is a clinical and laboratory disorder caused by
X-linked disruption of the purine metabolism. The deletion in the HPRT1 gene leads to the …

[引用][C] New personalized genetic mouse model of Lesch-Nyhan syndrome for pharmacology and gene therapy

PA Kusov, MI Yablonskaia, EN Korshunov… - 2018