Building precision medicine: Power to the patients

For precision medicine to be successful, we need to chip in. Here are some tools that patients can use to enable the growth of this field.

A new inlet to treating neurological disease

Researchers have found a way to control the blood-brain barrier to allow passage of small molecules, which could enable better treatment of CNS diseases.

A surprising new link between inflammation and mental illness — and a potential drug to protect the brain

Researchers have found a potential drug for protecting the brain from the neuropsychiatric effects of lupus and other central nervous system diseases.

Discovering a rare anemia in time to save an infant's life

"This is true precision medicine. We have been able to positively alter the course of a patient’s life, which is exactly what research efforts at a children’s hospital are meant to do."

Medical milestone: Making blood stem cells in the lab

A breakthrough from George Daley’s lab could allow patients’ own bone marrow cells to be genetically corrected and transplanted back into them, eliminating the need for a perfect donor match or toxic immune-suppressing treatments.

Custom-built ‘trainers’ help clinicians master procedures

These aren’t your traditional medical mannequins. To help them practice especially tricky procedures, our clinicians are teaming up with our Simulator Program to build custom, ultra-anatomically-correct “trainers.”

Featured Researchers + Innovators

  • Jean Connor, PhD, RN, CPNP
    Connor, who has her PhD in nursing, directs nursing research at the Boston Children’s Heart Center. Her work translates industry research into actionable lessons and innovations that improve care at the bedside.

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  • Katherine Janeway, MD
    “It’s all about the patients,” when asked about the motivations behind her efforts to bring precision medicine to pediatric oncology. But it’s more than that; the drive to combine science and care is in her blood. 

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  • Ken Mandl, MD, MPH
    Mandl is used to seeing the world through a different lens. In high school, he began clicking photographs and developing them in a darkroom in his basement. Now, he frames subjects through the lens of epidemiology and informatics.

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  • John Brownstein, PhD
    Boston Children’s Hospital’s new chief innovation officer is an epidemiologist by training and a founding father of the growing field of digital epidemiology—the use of digital data from a variety of sources to detect and track disease and promote health.

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  • Bruce Zetter, PhD
    Though he has had a lifelong passion for science, he once toyed with an acting career. But he stuck with science and pursued a career in academic medicine. Countless patients, students, business partners and mentees have benefitted.

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  • Michael J. Docktor, MD,
    Boston Children’s Hospital’s clinical director of Innovation and director of Clinical Mobile Solutions, is also a practicing gastroenterologist, a proud father of two and a passionate mobile-and-digital health trailblazer.

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  • Gena Koufos

    Gena Koufos, RN, MS, MBA, is program manager in Boston Children’s Hospital’s Innovation Acceleration Program. Her role entails designing new programs to support innovation acceleration across the institution

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  • David G. Hunter, MD, MPH

    David G. Hunter, MD, PhD, dreamed of a career as a rock star. Instead, he became Boston Children’s Hospital’s ophthalmologist-in-chief and invented the Pediatric Vision Scanner.

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  • Visner Lab
    Kaifeng Liu, MD, a research fellow at Boston Children’s Hospital, takes his inspiration from ants. Liu has taken this inch-by-inch approach in a radical redesign of the conventional suturing needle: “I started to play with the surgical needle in graduate school in 1986.”
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  • Martha Murray, MD has been on a 30-year quest to devise a better way to treat anterior cruciate ligament (ACL) tears.

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  • William Pu

    With all of the recent buzz about precision medicine, it’s no wonder that William Pu, MD is gaining recognition for his innovative application of stem cell science and gene therapy to study Barth syndrome, a type of heart disease that severely weakens heart muscle. Pu’s research was recently recognized by the American Heart Association as one of the top ten cardiovascular disease research advances of 2014.

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  • Daniel S.  Kohane, MD, PhD

    He’s a big thinker focused on harnessing the hyper-small. Daniel Kohane, MD, PhD, is a leading drug delivery and biomaterials researcher, leveraging nanoparticle technology and other new vehicles to make medications safer and more effective.

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  • Susan Faja, PhD

    Improbable as it sounds, autism researcher Susan Faja, PhD, likens her job to improv. “I really like Tina Fey’s description of her days as an improv comedian,” says Faja, who joined ...

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Featured Research Laboratories

  • Beggs Laboratory: Current studies are aimed at identification of new nemaline myopathy genes, understanding the basis for the variability observed, and determining how these mutations affect muscle function and lead to weakness.

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  • The Lencer Laboratory is located in the GI Cell and Developmental Biology Laboratories in the GI Division at Boston Children's Hospital.

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  • Sports Medicine Research Laboratory
    Led by principal investigator Dr. Martha M. Murray, focuses on sports medicine injuries, including those of the anterior cruciate ligament (ACL), knee meniscus and articular cartilage.

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  • Sankaran Laboratory
    Utilizing rare and common human genetic variation to improve our understanding of red blood cell production and globin gene regulation with application to numerous blood diseases.

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  • The Zon Laboratory focuses on the use of the zebrafish model for research into hematopoiesis and as a screen for oncogenic genes and proteins.

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  • The Gaab Laboratory
    Our multidisciplinary team of researchers brings together curious scientists from the basic and applied sciences, such as neuroscience, psychology, and education.
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