Our Approach

Approach Top Graphic


Approach 1aConnect with patients

New technologies in genome editing allow for the introduction of patient mutations more efficiently than ever before. The Jackson Laboratory works to understand the patient, the disease, and future therapeutic strategies. We work directly with Foundations & their Scientific Partners to ascertain the community’s needs and identify patient pathogenic variants to assess in mouse models.


Approach 2Anticipate Genetic Therapy
We engineer strategic models informed by variants, mechanisms, and potential therapeutic strategies. Several disease-modifying approaches, such as Antisense Oligonucleotide (ASO) Therapies and Gene Therapy, have successfully advanced into clinical application. The emergence of personalized therapies and small-scale individual drug trials is gaining momentum. We understand that various treatments necessitate different genetic designs in our mouse models. Our team, of dedicated scientists, is here to help design these models based on the future therapeutic approach, and we're eager to lend our experience, knowledge, and resources towards better model design.


Approach 3aEngineer Precision Models

Generating precise genetic mutation in mice is a critical step in preclinical drug development. Leveraging our extensive array of genome engineering technologies, we have the capability to produce a diverse range of precisely targeted mutagenesis models, encompassing humanized knock-ins, transgenic, and conditional models. The mission of the Jackson Laboratory is to make these models available for public distribution in the absence of restrictive agreements as soon as practicable, once standardized validation studies have been completed.


Approach 4

Characterize & Test Therapeutic Approaches

Much like a specialized mouse hospital, we possess a comprehensive suite of phenotyping capabilities, enabling us to validate and characterize mouse models for clinically relevant phenotypes. Collaborating closely with patients, families, and clinicians, we craft phenotyping pipelines with a focus on outcomes that translate directly to clinical practice. These measures serve as critical indicators of efficacy in our preclinical models.

For many rare disease patients, time is a precious and limited resource. With this in mind, our scaled preclinical services re-define the testing process. Rather than evaluating therapies one by one, we employ a parallel approach, accelerating the journey to advancements in treatment.


Approach 5aSupport Moving Therapies to the Clinic

Data is an asset. Generating precise models & conducting proof-of-concept preclinical work provides rare disease groups with essential tools to sustain research and promote drug development in their specific disease areas. This preclinical data, derived from disease models, serves as pivotal support for Investigational New Drug (IND) applications. It helps demonstrate the appropriate route of administration, dosing, & tolerability, laying a robust foundation for the advancement of potential treatments.