Church Describes Medical Potential of Gene Editing

Dr. George Church takes a question as NIH director Dr. Francis Collins looks on.
Dr. George Church takes a question as NIH director Dr. Francis Collins looks on.

The thought of transplanting animal organs into humans conjures up images of the chimera—the Greek mythological monster with a lion’s head and a goat’s body. While this extreme is a long way off, rapid progress in gene-editing techniques at the heart of realizing such a transplant hold promise for developing new diagnostics and therapies in the next few years.

Recently, genome sequencing and gene-editing techniques have become faster, more accurate and cheaper thanks to the innovations of investigators such as Dr. George Church, who recently delivered the Marshall Nirenberg Lecture in Masur Auditorium.

“The thing that’s significant here is that we’re not just reading genes and their 64 types of triplet codons, which comprise the genetic code, but we’re also now writing them and can do radical recoding,” said Church, professor of genetics, health sciences and technology at Harvard University and director, Harvard-NHGRI Center of Excellence in Genomic Science.

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Faster Sequencing, More Patients Boost Autism Research

A philanthropist’s largesse and faster sequencing technology are helping researchers gain footholds in understanding autism spectrum disorder, which affects between 1 and 2 percent of the population.

But complex diseases of brain and behavior such as ASD will not yield soon to simple solutions, said Dr. Matthew State, chair of the department of psychiatry at the University of California, San Francisco, School of Medicine.

ASD is “a tremendously heterogeneous disorder,” he said at a recent NHGRI intramural seminar in Lipsett Amphitheater. “Anyone who comes to us and says that 10 out of 10 autism patients have the same marker for idiopathic disease is [likely misguided].”

Genetic surveys of the cause of ASD began to be successful in the early 2000s, he said, with research groups now converging on roughly the same set of genes during the past decade. Some 65 ASD-associated genes have been identified so far, said State, with scientists having the highest confidence in about 30 of them.

“We expect the final number will be somewhere between 400 and 1,000,” he said.

It has long been known that genes play a major role in ASD, a broad set of disorders of social communication, but State said there are no treatments that are highly effective, particularly for the core social deficits. “The mainstay, first-line treatment is behavioral therapy,” he noted. “But our lack of knowledge about the molecular, cellular and circuit mechanisms in ASD is profoundly limiting.”

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