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Downstate Researchers Identify New Molecular Driver of Breast Cancer, Opening Door to Novel Therapies

By Office of Communications & Marketing | Apr 21, 2026

Brooklyn, NY — Scientists at SUNY Downstate Health Sciences University (Downstate) have uncovered a previously unknown molecular mechanism that promotes breast cancer development, offering a promising new target for future treatments.

In a study published in iScience, Downstate researchers demonstrate that a regulatory RNA molecule, known as BC200 RNA, an drive breast cancer by repressing the tumor-suppressor gene BRCA1, a key player in DNA repair and cancer prevention.

“BRCA1 has long been recognized as one of the most important genes in protecting against breast cancer,” said senior author Henri Tiedge, Ph.D., distinguished professor at Downstate. “Our findings reveal a completely new way that BRCA1 function can be disrupted—not by mutation, but by regulatory RNA interference.”

This publication reflects a deeply integrated collaboration between SUNY Downstate and Mirimus Inc., bringing together academic expertise in RNA biology with advanced genetic engineering technologies to enable mechanistic discovery.

“By combining Downstate’s leadership in RNA biology with Mirimus’ expertise in genetic engineering and RNA interference technologies, this collaboration highlights how academic and biotech teams can work together to uncover new disease mechanisms and enable future therapeutic strategies,” said Prem Premsrirut, M.D., Ph.D., President and CEO of Mirimus.

A New Mechanism Behind Breast Cancer

The study shows that BC200 RNA, normally active only in neurons, becomes abnormally expressed in breast epithelial cells. Once present, it blocks the production of the BRCA1 protein, leading to:

  • Increased DNA damage
  • Loss of normal cell repair mechanisms
  • Accelerated tumor growth and malignant transformation

Researchers also demonstrated that introducing BC200 RNA into healthy breast cells was sufficient to transform them into malignant cancer-like cells, underscoring its causal role in disease development.

Reversing Cancer Behavior

Importantly, the team found that reducing BC200 RNA levels using RNA-targeting approaches reversed cancer-like behavior in cells:

  • Restored BRCA1 expression
  • Reduced DNA damage
  • Significantly decreased tumor growth and cell proliferation

“These results suggest that BC200 RNA is not just involved in cancer—it may be a powerful therapeutic target,” said lead author Valerio Berardi, Ph.D., assistant professor at Downstate.

What This Means for Patients

This discovery expands how clinicians understand breast cancer risk and treatment. It shows that BRCA1 function can be disrupted even without a gene mutation, helping explain why some patients develop aggressive disease without known genetic risk factors.

The findings also point to a new treatment strategy of targeting BC200 RNA to restore normal BRCA1 function. This approach may lead to more precise therapies, particularly for patients who do not respond to current treatments.

Linking the Nervous System to Cancer

The research also uncovered a surprising connection between the nervous system and breast cancer. The team found that nerve growth factor (NGF), a molecule involved in nerve signaling, can trigger BC200 RNA expression, linking tumor innervation to cancer progression.

“This research provides new insight into how the tumor microenvironment, including nerves, may actively contribute to cancer development,” said David Christini, Ph.D., Senior Vice President for Research at Downstate.

Implications for Future Treatments

Current therapies targeting BRCA1-related cancers often exploit weaknesses in DNA repair. This study suggests an alternative strategy of restoring BRCA1 function by targeting BC200 RNA upstream.

While further validation in animal models and clinical settings is needed, the findings position BC200 RNA as a promising candidate for next-generation breast cancer therapies.

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About Mirimus

Mirimus is a biotechnology company focused on advancing precision genetic engineering and in vivo disease modeling to accelerate the development of next-generation therapeutics. The company leverages proprietary platforms, including CRISPR-based technologies and RNA-targeting approaches, to generate sophisticated animal models that enable a deeper understanding of disease biology and more predictive preclinical research. Mirimus partners with academic institutions, pharmaceutical companies, and research organizations to translate scientific discoveries into impactful therapeutic strategies.

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About SUNY Downstate Health Sciences University

Downstate Health Sciences University is Brooklyn’s academic medical center, the only SUNY academic medical center in New York City. It serves as a critical hub for healthcare delivery, medical education, and research for Brooklyn’s 2.7 million residents and expanded care access.

University Hospital at Downstate (UHD) delivers high-acuity and community-based care across the borough, with more than 800 physicians across 53 specialties and subspecialties. UHD provides high-risk neonatal and infant care, pediatric nephrology, and dialysis services, and established Brooklyn’s first kidney transplantation program more than 50 years ago.

Downstate strengthens the healthcare workforce and advances community-based care through its academic programs and clinical training. Its biotechnology initiatives, including the Biotechnology Incubator and BioBAT, drive innovation and economic growth across Brooklyn and New York City.