New from CGM in Science Translational Medicine: CGM's Silva, Morini & team developed small molecules that fix a MAPT splicing error, lowering toxic 4R tau in FTD neurons and a mouse tauopathy model. A promising step toward FTD-tau therapeutics. doi.org/10.1126/scitranslmed.ady6759

MAPT splicing modulators reduce 4R tau and rescue tauopathy phenotypes in human neurons and in a mouse model

MAPT splicing modulator compounds reduce 4R tau and rescue pathology in neurons derived from patients with tauopathy and mouse models.

doi.org

Yesterday's CGM Seminar featured Rakesh Karmacharya and members of his lab, who are working at the intersection of clinical psychiatry and stem cell biology to understand schizophrenia and bipolar disorder.

Congratulations to Emily King, PhD (mentored by @bkleinstiver.bsky.social) who successfully defended her PhD thesis last week! The broader goal of her work is to shorten the path between technology development and therapeutic translation.

Congratulations to CGM Faculty Pradeep Natarajan (pnatarajanmd.bsky.social) who was recently promoted to Full Professor of Medicine at Harvard Medical School!

🧬 Happy DNA Day from the Center for Genomic Medicine! 🧬 National DNA Day is celebrated annually on April 25th to commemorate the 1953 discovery of DNA's double helix structure and the 2003 completion of the Human Genome Project. Let us know how you're celebrating!

This week CGM, Ricardo Mouro Pinto, PhD, shared new insights into repeat expansion disorders. His work highlights somatic repeat expansion as a key driver of disease—and a promising target for biomarkers and therapeutic intervention

Two of our CGM trainees were recognized as Posters of Distinction at the inaugural 2026 MGB Celebration of Science! Congratulations to Dr. Ieva Miceikaite (Talkowski Lab) and Dr. Juan De la Hoz (Ge and Smoller Labs) on this remarkable accomplishment

Great talk by Wendy Chung (Boston Children's Hospital): “Mendelian Diseases: Too Rare to Care?” Rare diseases are individually rare but collectively common (>50% of pediatric inpatients). Shared biology + connected communities accelerate progress. Early diagnosis = earlier, more effective treatment.

Congratulations again to CGM faculty Jordan Smoller and Ben Kleinstiver on their Martin Prizes! Today, at the MGB Celebration of Science, they received their awards and delivered insightful talks on their prize winning papers. The entire CGM community is thrilled for both of them.

Today, we welcomed Merit Cudkowicz, MD, MSc, Executive Director of the MGB Neuroscience Institute (NSI) and Craig Blackstone, MD, PhD, Scientific Director of the NSI, to our Genomic Medicine Seminar Series to give us a look at the NSI's goals, priorities, future directions, and early successes.

Today, the CGM had the pleasure of welcoming Dr. Evan Eichler, PhD, (eichlerlab.bsky.social) to our Genomic Medicine Seminar series. His enthusiasm for his research was apparent and inspiring. Thank you, Dr. Eichler, for your visit and for infusing our day with such vibrant energy!

On this #WayBackWednesday, we’re revisiting last week’s Genomic Medicine seminar featuring Dr. Po-Ru Luh from the Brigham and Women's Division of Genetics! We at CGM have been enjoying familiarizing ourselves with the science from our BWH colleagues.

Congratulations to CGM and Psychiatry Faculty Dr. Jordan Smoller (@jorsmo.bsky.social), winner of the 2026 Martin Prize in Population Health Sciences!

Please join us in congratulating our new Associate Director, Raghu Chivukula! We are excited to partner with him as we continue advancing the CGM mission, facilitating the scientific goals of our scientific and administrative teams, and building on the CGM’s unparalleled genomic medicine community.

Last week, Dr. Mike Talkowski, CGM Director, met with trainees and faculty in the Division of Genetics at Brigham and Women’s Hospital. We're thrilled that his seminar, Dr. Cole's last month, and Po-Ru Loh's today at CGM are connecting our MGB Genomics community.

At today's seminar, we heard from Dr. Alexandre Reymond, Visiting Professor in the Center for Genomic Medicine and Broad Institute. We loved hearing about his groundbreaking work and are delighted he's spending time with us in CGM!

Today is Rare Disease Day, highlighting the millions affected by rare genetic conditions. At the Center for Genomic Medicine, today and always, we honor the resilience of the rare disease community and reaffirm our commitment to impactful science.

The snow couldn't stop the Samocha lab from presenting at CGM seminar today. We heard from @ksamocha.bsky.social, her lab members Katherine Chao (gnomAD Product Manager) and Yitang Sung (Postdoctoral Fellow) about their work.

No CGM Seminar today so let’s revisit last Wednesday when Dr Jordan Smoller, Dr Travis Mallard, & Dr Justin Tubbs discussed their work on Psychiatric genomics, progress in psychiatric genetics, the changing diagnostic system & the genetics of Externalizing Disorders & Self-Reported Psychopathy

Happy International Day of Women & Girls in Science! We love any excuse to celebrate women and girls in science. Especially our tremendous female faculty and associate members!

Excited to announce that applications are open for the Mass General Brigham T32 Postdoctoral Training Program in Precision & Genomic Medicine!

CGM's Martin, He & team discovered 25 new genetic variants for lung function in East Asian populations & created a polygenic risk score that better predicts asthma, COPD & lung cancer across diverse groups. https://loom.ly/yj_l-Oo

Exciting breakthrough from CGM Faculty Dr. Vamsi Mootha and colleagues describing a potential new therapeutic path for Friedreich's ataxia, a devastating genetic disorder that typically strikes in childhood and dramatically shortens lives. https://loom.ly/JxV0-gc

The McCance Center kicks off its 2026 Seminar Series next month with Ana Griciuc, PhD, on February 9th at 11 am. Don’t miss out on the insights from industry leaders and innovators throughout the year! More speakers to come! Learn more: massgeneral.org/neurology/mccance-center/events

We were so thrilled to host kiranmusunuru.bsky.social as part of our Genomic Medicine Seminar Series! It was a day full of productive meetings with colleagues, an exciting hour-long talk on his groundbreaking work in CRISPR-based therapeutics, and a trainee roundtable discussion.

Join us for an exciting CGM seminar today at noon with the Children's Hospital of Pennsylvania's very own kiranmusunuru.bsky.social (also a CGM alum). You don't want to miss it!

Join us for an exciting CGM seminar today at noon with the Children's Hospital of Pennsylvania's very own @kiranmusunuru.bsky.social (also a CGM alum). You don't want to miss it!

Another great seminar this week with Philip A Cole, MD, PhD from BWH Genetics. We're so grateful he came over to give the first of many talks featuring speakers from across our MGB genetics and genomics community.

New work from the Wheeler lab shows DNA repair gene MSH3 modifies disease: variants that boost repeat expansion cause earlier onset, while those promoting contraction delay onset—in blood and brain. Targeting MSH3 could delay or prevent X-Linked Dystonia Parkinsonism (XDP)

Our newest CGM faculty member, @alicestanton.bsky.social, recently published this exciting paper in PNAS titled, "Engineered 3D immuno-glial-neurovascular human miBrain model". The paper describes her innovative work creating the MiBrain platform.