A devastating disease that robs the elderly of their memories seems a world away from rare genetic diseases that set children on a path of pain, deformity, and even early death. But scientists are focusing on a common thread: an intricate origami gone awry in cells.
Graphic: Cell origami
Normally, cells make proteins that are folded into various three-dimensional shapes and ferried to different areas of the cell to do their work. Those jobs, ranging from fighting infections to helping orchestrate cell processes, are essential to the life of the cell. But sometimes, because of genetic mutations or reasons scientists still don’t fully understand, key proteins crumple into the wrong shape.
That simple mistake can result in anything from emphysema to cancer to mad cow disease. In some diseases, such as cystic fibrosis, misfolded proteins aren’t transported to the part of the cell where they are critically needed. In others, misfolded proteins clump into toxic tangles like the ones found in the brains of Alzheimer’s patients.
Now, a growing slate of Cambridge companies have joined the search for drugs that could correct, clear away, or stabilize misfolded proteins and reverse the damage they cause.
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A devastating disease that robs the elderly of their memories seems a world away from rare genetic diseases that set children on a path of pain, deformity, and even early death. But scientists are focusing on a common thread: an intricate origami gone awry in cells.
Graphic: Cell origami
Normally, cells make proteins that are folded into various three-dimensional shapes and ferried to different areas of the cell to do their work. Those jobs, ranging from fighting infections to helping orchestrate cell processes, are essential to the life of the cell. But sometimes, because of genetic mutations or reasons scientists still don’t fully understand, key proteins crumple into the wrong shape.
That simple mistake can result in anything from emphysema to cancer to mad cow disease. In some diseases, such as cystic fibrosis, misfolded proteins aren’t transported to the part of the cell where they are critically needed. In others, misfolded proteins clump into toxic tangles like the ones found in the brains of Alzheimer’s patients.
Now, a growing slate of Cambridge companies have joined the search for drugs that could correct, clear away, or stabilize misfolded proteins and reverse the damage they cause.
To Read This Article In It’s Entirety: Click Here