Improving With Age

Aging is inevitable. It touches everyone. With the passing of time comes the greater and inevitable likelihood of diminishment and age-related disease. 

By age 55, for example, 78 percent of American adults will have at least one chronic condition; by age 65, that number grows to 85%, with almost one-quarter reporting three or more chronic ailments.

Aging and its consequences affect every part of our well-being and lives, from single cells to whole systems. We seek to understand how aging works and why, and with that knowledge find new ways to slow its progress and improve the prospects of longer, healthier lives.

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News

Helping Ourselves

The ability to fend off infectious diseases, which represent a mortal threat to millions, rests with the body’s immune system, which provides protection through various, layered and complex defenses. 

If the immune system fails to function properly, if it becomes too weak, it can increase susceptibility to infectious disease. If it becomes too strong or dysfunctional, it can result in  harmful inflammation and autoimmune diseases such as lupus and arthritis and even cancer.

By studying pathogen-host interactions, innate and humoral immunity, inflammation and T cell checkpoint regulation at even the smallest of interactions, we seek to better understand how the immune system works, why it sometimes doesn’t and find new treatments for endemic and pandemic infectious diseases, autoimmune disorders, cancer and inflammatory conditions. 

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News

Everything Stems Here

Stem cells are the body’s source of raw materials. These are the cells from which all other cells with specialized functions, such as immune or neurological, are generated. Researchers can now create and differentiate stem cells in the lab with induced pluripotent stem cells (iPSCs).

We use iPSCs to probe the origins and processes of disease, to deepen understanding of developmental biology and what it means to be healthy or recover from injury and illness. This basic knowledge helps drive the engines of discovery and therapeutic solutions. 

Evan Snyder, MD, PhD
Director

An Uncommon Concern

A “rare disease” is a condition afflicting less than 200,000 people in the U.S. In sheer numbers, however, they are not so rare. Scientists have documented more than 7,000 affecting approximately 30 million Americans. 

Thanks to immunizations and improving health care, most childhood diseases are uncommon. But rarity can be a curse. The most difficult aspect of being stricken by a rare disease may be the difficulty in getting a correct diagnosis. Rare diseases are frequently misdiagnosed or undiagnosed. On average, patients with rare diseases wait nine years to finally receive an accurate diagnosis, a figure that hasn’t changed in 45 years. Half of all rare disease cases involve children, of which one-third will die before they are 5 years old, before doctors even figure out what killed them.

Our mission is to advance research and understanding of diseases that predominantly affect children, which are under-addressed or ignored altogether. These include diseases such as diabetes, Duchenne muscular dystrophy, hypophosphatasia (bone disorders) and congenital disorders of glycosylation.

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Symposium

Rare Disease Day

Rare Disease Day is an international initiative occurring on the last day of February to raise awareness of rare diseases as a significant public health concern. Every year Sanford Burnham Prebys invites scientists, physicians, patients, families and advocates to highlight progress, exchange ideas and concerns on selected disorders. This year’s focus is on congenital disorders of glycosylation, an umbrella term that covers more than 190 rare (fewer than 2,000 children worldwide) genetic diseases involving a complex chemical process called glycosylation.

When this metabolic process, which involves attaching essential sugar molecules to proteins, doesn’t work correctly, numerous, often severe, consequences in infancy and childhood occur, from poor growth and developmental delays to stroke-like episodes and heart problems.

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Leadership

Director

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News