Science

Tools For Researchers

We are partnering with The Jackson Laboratory (JAX) to provide researchers with reliable tools for studying PURA Syndrome including a newly developed Knockout PURA Mouse Model and Conditional Knockout PURA Mouse Model. In addition, we have partnered with JAX to create induced pluripotent stem cell (iPSC) lines.


PURA Mouse Models

These PURA Mouse Models were recently featured by the American Society of Gene & Cell Therapy (ASGCT) at their annual meeting, the largest and most respected gathering of researchers and industry leaders working in gene and cell therapy.

PURA-KO Mouse Model

PURA-KO Mice have exon 2 deleted in the PURA gene. These mice may be useful when studying regulatory roles of PUR-α in DNA replication, gene transcription, RNA transport, and mRNA translation.

Click below for further information about this mouse model and instructions on how to order today.

PURA-cKO Mouse Model

PURA-cKO Mice have loxP sites flanking exon 2 of the PURA gene. These mice may be useful for generating conditional mutants when studying regulatory roles of PUR-α in DNA replication, gene transcription, RNA transport, and mRNA translation.

Click below for further information about this mouse model and instructions on how to order today.


PURA iPSC Lines

JAX has created several iPSC lines that can be used to test therapeutics that affect PURA Syndrome’s main symptoms on brain function and motor control.

Engineered Induced Pluripotent Stem Cell Lines

There are 9 iPSC lines available from JAX, you can find them all by clicking below to visit JAX’s iPSC Catalog and typing "PURA" in the search bar.

You'll see the 3 mutations and if you expand each result you can see the 3 lines for each mutation (het, hom and wt/reverant).


Funding Policy

Jack's Tomorrow recognizes that grant award recipients may have overhead costs that are not directly attributable to the proposed project but are necessary to carry out the proposed project.  Jack's Tomorrow will not fund indirect costs, and therefore can only make grants to researchers and institutions that can absorb the indirect costs of the project. 

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Your support directly helps us fund important work like this.

Consider a donation today and join the Jack’s Tomorrow team as we find and fund a cure for PURA Syndrome.