Patti Dickson
@pattidickson.bsky.social
Medical geneticist, mom, wife. Researching lysosomal diseases. Division Chief at Washington University in St. Louis, Missouri. UDN, IGVF, PE-CGS, GIC, COMBINE. Opinions, likes, and follows do not represent WashU
When we restricted restoration of the corrective enzyme to brain neurons, brain HS normalized as did CSF HS, without impacting HS levels in the bloodstream. We interpret the results of these murine experiments as suggesting that CSF HS reflects HS in the brain in neuronopathic MPS disorders.
November 28, 2025 at 7:57 PM
When we restricted restoration of the corrective enzyme to brain neurons, brain HS normalized as did CSF HS, without impacting HS levels in the bloodstream. We interpret the results of these murine experiments as suggesting that CSF HS reflects HS in the brain in neuronopathic MPS disorders.
We designed a set of mouse experiments to determine whether reducing heparan sulfate systemically, including in the bloodstream, might cause the reduction in CSF heparan sulfate. We restricted corrective enzyme to the body and found that lowering blood HS had no effect on CSF HS.
November 28, 2025 at 7:57 PM
We designed a set of mouse experiments to determine whether reducing heparan sulfate systemically, including in the bloodstream, might cause the reduction in CSF heparan sulfate. We restricted corrective enzyme to the body and found that lowering blood HS had no effect on CSF HS.
We had observed that CSF heparan sulfate decreased in MPS I patients treated with enzyme replacement therapy, despite that ERT is thought not to cross the blood brain barrier.
November 28, 2025 at 7:57 PM
We had observed that CSF heparan sulfate decreased in MPS I patients treated with enzyme replacement therapy, despite that ERT is thought not to cross the blood brain barrier.
People are doing some great work.
October 17, 2025 at 11:17 PM
People are doing some great work.
Third, since 1 in 10 people have a rare disease, chances are you or someone you care about is impacted by them, so why would you want to discourage folks from working on them?
August 15, 2025 at 2:06 PM
Third, since 1 in 10 people have a rare disease, chances are you or someone you care about is impacted by them, so why would you want to discourage folks from working on them?
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grants.gov
June 5, 2025 at 9:30 PM
Congratulations Susan!
May 29, 2025 at 7:28 PM
Congratulations Susan!
Not the same. You have to think from our perspective about the types of scenes we often have to watch, the positions we are put in, the roles we have in advancing the narrative. This was a show where women had power really in every situation (even when cut off from the source).
May 23, 2025 at 8:45 PM
Not the same. You have to think from our perspective about the types of scenes we often have to watch, the positions we are put in, the roles we have in advancing the narrative. This was a show where women had power really in every situation (even when cut off from the source).
I did not read the books, but I loved the show. They never put a woman in a situation where she was powerless.
May 23, 2025 at 8:36 PM
I did not read the books, but I loved the show. They never put a woman in a situation where she was powerless.