CRISPR for Rare Disease and AAV Vector Platform



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

  1. Knockout and knock in services in mouse and rat

  2. Humanization: in situ replace mouse gene with matched human exons or introns

  3. Transgenic, genotyping and phenotyping

  4. In vitro fertilization

  5. Embryo and sperm cryopreservation

  6. Drug administration, animal and tissue manipulation and histopathology

  7. AAALAC accreditation and GLP compliance


General Adeno-associated Viral Vector (AAV) Platforms

  1. Customized recombinant AAV (rAAV) genome construction and validation

  2. General AAV packaging, purification and tittering

  3. Premade AAV control vectors in vitro and in vivo transduction


AAV vector development

  1. Customized rAAV vector design, construction and packaging

  2. In vitro validation

  3. Dose-escalation long-term efficacy and safety evaluation in vivo including wild type and disease models


AAV-mediated in vivo genome editing

  1. Customized rAAV vector design, construction and packaging for in vivo genome editing

  2. In vitro validation of genome editing

  3. In vivo genome editing efficiency and functional evaluation