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PouLab team wins NIH CRISPR Challenge!

 

We’re thrilled our team won Phase 2 of the NIH TARGETED Challenge (Targeted Genome Editor Delivery)!!! Suffice it to say, several rounds of celebrations ensued.

Our entry, Crisaptics Trans-BBB, competed in Area 2: Genome Editing in Brain Across the Blood-Brain Barrier, an exciting competition drawing entries from across academia, biotech, and research institutes, as part of the Challenge initiated by the NIH Common Fund and its Somatic Cell Genome Editing (SCGE) Consortium.

Crisaptics Trans-BBB merges advanced in vivo genome editing using Cas9-RC together with new delivery technologies enabled by BBB-penetrating nanoparticles and focused ultrasound. With these emerging tools, we were able to edit the genome of the brain in spatially targeted areas without any invasive procedure. This milestone is a critical step toward our quest to develop genome therapeutics for the brain.

The project received $250,000 in discretionary funding and an invitation to compete in Phase 3, where we’ll demonstrate editing in larger models. This award accelerates our mission to develop genome therapeutics for glioblastoma, Huntington’s disease, and genetic epilepsies

The Crisaptics Trans-BBB team is a four-lab collaboration within UM-MIND:

Poulopoulos Lab – in vivo CRISPR genome editing
Suk Lab – brain-penetrant nanoparticle bioengineering
Anastasiadis Lab – preclinical focused ultrasound
Woodworth Lab – clinical neurosurgery and therapeutic ultrasound

Learn more about the Challenge and the other winners:
NIH Announces Phase 2 Winners of the TARGETED Challenge

Crisaptics

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Colin’s Prime editing in neurons paper out in Methods Mol Biol!

Congrats to Colin Robertson and other lab members for publishing “Prime Editing of Mouse Primary Neurons” on how to use Prime editors on neurons to introduce precise genome edits, including pathological variants from patients.

This paper also includes a step-by-step guide on how to use our web app for pegRNA design and cloning PegAssist.app!

We should be hearing more exciting news from Colin and this powerful new technology for neural somatic genome editing soon…!

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PouLab Helps Host GBSfN 2024!

The annual reunion of Baltimore Neuroscientists, the Greater Baltimore Society for Neuroscience (GBSfN) meeting, took place this year in Westminster Hall (the famous resting place of Edgar Allan Poe) on the University of Maryland campus in downtown Baltimore, and the lab was front and center! Alex served as GBSfN President this year, and all lab members pitched in with logistics and science to make this an awesome day for Baltimore Neuro!

Over 170 members (from undergrads to Institute Directors) across our 9 academic institutions, with representatives from government and industry partners came together to celebrate Baltimore Neuroscience. We honored Baltimore icons like Vernon Mountcastle, Sol Snyder, and Linda Richards; discussed the evolving neuroscience technology toolkit with guest speaker George Church; and topped it all off with food, drinks, posters, and neuro-nerd bar trivia. What a day! Thank you to all that made it possible…

 

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JaNýa n’ Team Win Grand Prize in Case Challenge ’24 at the National Academy of Medicine!

Congratulations to JaNýa Brown, who’s team won the grand prize at the 2024 D.C. Public Health Case Challenge at the National Academy of Medicine!!! Their proposal “Guiding Resilience and Offering Wellness (GROW)” came in at the top of this year’s competition on “A Public Health Approach to Address Substance Use and Mental Health Concerns Among Emerging Adults in the DMV Area”. Read all about it in the linked press release by the NAM!

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Neuroligin-3 paper gets the cover of Biological Psychiatry!

Biol Psychiatry cover, Nov 2024

Congrats to Bek Altas and other lab members, as well as our collaborators at the Max Planck Institute for Multidisciplinary Sciences and the University of Turin. Our paper on the synaptic localization of Neuroligin-3 in the mouse and human brain and the molecular mechanism that regulates it, became the cover article on this month’s issue of Biological Psychiatry! Congrats to Cheryl Brandenburg for the cover art and the epic experiment that inspired it, one of the first ever immunolabelings of excitatory and inhibitory synapses in the human brain!

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Two collaborative papers just out on new methods for in vivo CRISPR delivery!

The first study, led by the Mackis Lab at Harvard University and featured in Cell Reports, develops an approach for delivering genes, including Cas9, to select neural populations by in utero electroporation. This method achieves internally-controlled mosaicism that facilitates phenotype discovery in the developing brain.

The second paper, from the Suk Lab at UMSOM and published in ACS Nano, demonstrates our ability to target Cas9 mRNA to select foci in the adult brain. This was achieved through systemic delivery using lipid nanoparticles, coupled with localized targeting by microbubble-mediated focused ultrasound. This non-viral strategy represents a significant advance in treating adult brain disorders by enabling focal genome editing directly in the adult brain.

Together, these studies represent substantial progress in non-viral CRISPR delivery methods for both developing and adult brains. By expanding the in vivo CRISPR toolkit, we are laying the groundwork for the development of neural somatic cell genome editing as future genomic therapeutics.

Congratulations Luciano and Gijung!

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Presenting Dr. Colin Robertson, PhD!

Colin Robertson becomes the 3rd member of the lab to defend their PhD, in the historic Davidge Hall, the longest serving Medical Lecture Hall in America!

Colin defended his pioneering work in using Prime Editing, a reverse-transcriptase hybrid CRISPR technology, in the developing mouse brain to model individual patients with genetic epilepsies. Colin is staying on as a postdoc to translate these CRISPR technologies into clinical applications.

Congratulations Colin!!