CRISPR in vivo for model generation and rare genetic disease
1.From genome editing to disease models and PK-PD-efficacy studies
2.Well-established in vivo gene editing platform in rodent
3. 100+ KO and KI mouse/rat lines generated
4.Generation of genetically engineered animal models (GEAM) for drug discovery for rare genetic disease
GEAM-based services
Knockout and knock in services in mouse and rat
Humanization: in situ replace mouse gene with matched human exons or introns
Transgenic, genotyping and phenotyping
In vitro fertilization
Embryo and sperm cryopreservation
Drug administration, animal and tissue manipulation and histopathology
AAALAC accreditation and GLP compliance
General Adeno-associated Viral Vector (AAV) Platforms
Customized recombinant AAV (rAAV) genome construction and validation
General AAV packaging, purification and tittering
Premade AAV control vectors in vitro and in vivo transduction
AAV vector development
Customized rAAV vector design, construction and packaging
In vitro validation
Dose-escalation long-term efficacy and safety evaluation in vivo including wild type and disease models
AAV-mediated in vivo genome editing
Customized rAAV vector design, construction and packaging for in vivo genome editing
In vitro validation of genome editing
In vivo genome editing efficiency and functional evaluation