Category Archives: Biotech

Science Fiction Becomes Science-Fact: Two Strategies for Repairing Humans

Here’s my first article in a series for Slate magazine on longevity. Thanks to Prudential for sponsoring my obsession with health extension!

“Not long ago, it would have sounded like science fiction to discuss growing human organs in the lab or re-writing DNA. Yet today both are realities that will change the world and allow for longer and healthier lives.

Already, lab-grown bladders, windpipes and blood vessels have been successfully created and implanted into humans. Most recently, tissue engineering pioneer Dr. Anthony Atala and his team at the Wake Forest Institute for Regenerative Medicine announced another breakthrough: lab-made vaginas—one of the most complex organs made to date. In four girls with MRKH syndrome, a medical condition in which the vagina and uterus are underdeveloped or absent, Dr. Atala’s team was able to create new organs that functioned normally, dramatically increasing each patient’s quality of life.

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Read more here.

Biologists Create Cells With 6 DNA Letters, Instead of Just 4

The Scripps Research Institute (TSRI) has just announced that it has created cells with an expanded genetic alphabet — an X and a Y added to the regular ACTG of DNA. This is a huge win for the field of synthetic biology. Here’s the Wired story.

“We now have a cell that survives and lives with more information in its genome,” said Floyd Romesberg, the synthetic biologist at the Scripps Research Institute in La Jolla, California who led the work.

Having more letters to work with potentially opens the door to a huge range of novel molecules. (A rough analogy: Just think how many crazy new words you could spell with 39 letters instead of the usual 26). With further refinements, synthetic cells might one day be used to create–or evolve–proteins that don’t exist in nature, as well as new sequences of DNA and RNA, any of which could be useful for research, diagnosing disease, or creating new therapies. But that’s still a ways off.”

RMF Conference — photo with Dr. Atala

I was honored to be on a panel tonight with tissue engineering pioneer Dr. Anthony Atala, Patient advocate Katie Jackson, and science artist Kelly Milukas. It was a fun panel and a great discussion about how to get more community members involved in supporting life-saving advances in regenerative medicine. Our audience, mainly scientists, wowed me after the discussion with stories of their world-changing work.
Atala

Stem cells used to repair animal hearts and human muscle

More good news from the regenerative medicine scene. One of the studies was conducted by Dr. Stephen Badylak, whose work was profiled in my book. Here’s the story from USA Today:

Two new studies out today show both the incredible promise of stem cell research and its current limitations.

In one, published in the journal Nature, researchers showed that they could repair damaged hearts by injecting these versatile stem cells into macaque monkeys. Heart disease is the leading cause of death, and if the same process can work in people, it could benefit hundreds of thousands a year.

In the other study, published in Science Translational Medicine, five men were able to regrow leg muscles destroyed by accidents or military service. The researchers, from the University of Pittsburgh, inserted into the men’s muscles a “scaffold” of muscle tissue from a pig. Through aggressive physical therapy right after the surgery, the men’s own stem cells were encouraged to populate the scaffold and substantially rebuild their leg muscles.

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Another win for organ-growing

Dr. Anthony Atala and his team at Wake Forest recently announced yet another organ-growing success, this time more complicated than before. As the WSJ reports:

Scientists have successfully transplanted laboratory-made vaginas into four teenage girls whose own were absent because of a rare disease, marking a milestone in the quest to grow structurally complex body parts.

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Some of the easiest organs to make are flat structures, like skin, typically composed of a single type of cell. Tubular body parts including windpipes, urethras and blood vessels are harder to fashion as some are typically made of two cell types. But in recent years several such organs have been successfully transplanted into patients.

Another step up in complexity are body parts such as the vagina, a highly elastic organ that also secretes mucus. And the final frontier is the quest to make solid organs—such as the heart and liver—that typically don’t have a cavity and have more complex functioning.

New genomics initiative at UC Berkeley

With a $10 million gift from the Li Ka Shing Foundation, Berkeley recently announced the formation of the Innovative Genomics Initiative (IGI). Its purpose is to “lead a revolution in genetic engineering based on a new technology already generating novel strategies for gene therapy and the genetic study of disease.”

Excellent. And UC Berkeley is a good place to do it since the university is home to Dr. Jennifer A. Doudna, who is credited with co-discovering CRISPR/Cas9, precision “DNA scissors” that allow scientists to better edit genes.

Scientists regenerate immune organ in mice

UK scientists report that they have fully restored a degenerated organ in a living animal, a discovery that could pave the way for future human therapies.

Professor Clare Blackburn from the MRC Centre for Regenerative Medicine, at the University of Edinburgh, who led the research, said: “By targeting a single protein, we have been able to almost completely reverse age-related shrinking of the thymus. Our results suggest that targeting the same pathway in humans may improve thymus function and therefore boost immunity in elderly patients, or those with a suppressed immune system. However, before we test this in humans we need to carry out more work to make sure the process can be tightly controlled.”

Here’s a BBC article about the work. And the original press release.

Bio-engineer predicts a bio-printed heart in a decade

From Wired UK:

“America put a man on the Moon in less than a decade. I said a full decade to provide some wiggle room,” Stuart K Williams told Wired.co.uk.

Williams is heading up the hugely ambitious project as executive and scientific director of the Cardiovascular Innovation Institute at the University of Louisville. Throughout his prestigious career spanning four decades he has focused on researching surgical devices and bioengineering, and the idea for printing the heart whole from scratch was inspired by the work of one of the pioneers in both these fields — Charles Lindbergh. Lindbergh might be best known for flying solo across the Atlantic and for the Crime of the Century (when his infant son was kidnapped and murdered) but he also created a glass perfusion pump with Alexia Carrel that would keep the human heart alive outside the body, paving the way for heart surgery. The pair also discussed regenerative medicine in their book The Culture of Organs.

Blood vessel cells can repair organs, say Weill Cornell scientists

Another great bioengineering study. This one showing the possibility of repairing organs simply by injecting them with engineered endothelial cells.

Here’s the press release:

Damaged or diseased organs may someday be healed with an injection of blood vessel cells, eliminating the need for donated organs and transplants, according to scientists at Weill Cornell Medical College.

In studies appearing in recent issues of Stem Cell Journal and Developmental Cell, the researchers show that endothelial cells — the cells that make up the structure of blood vessels — are powerful biological machines that drive regeneration in organ tissues by releasing beneficial, organ-specific molecules.

They discovered this by decoding the entirety of active genes in endothelial cells, revealing hundreds of known genes that had never been associated with these cells. The researchers also found that organs dictate the structure and function of their own blood vessels, including the repair molecules they secrete.

Together, the studies show that endothelial cells and the organs they are transplanted into work together to repair damage and restore function, says the study’s lead investigator, Shahin Rafii, M.D., a professor of genetic medicine and co-director of the medical college’s Ansary Stem Cell Institute and Tri-SCI Stem Center. When an organ is injured, its blood vessels may not be able to repair the damage on their own because they may themselves be harmed or inflamed, says Dr. Rafii, who is also an investigator at the Howard Hughes Medical Institute.

A step on the way to therapeutic brain cells?

Will a person’s own brain cells be used to repair their brain one day? Maybe. Here’s an article that discusses the potential first steps — taking brain cells from a living person and growing more of them.

From the Third Age:

Researchers at the University of Western Ontario have found that cells from brain biopsies can be used to grow large numbers of patient’s own brain cells. These new therapeutic cells, when reintegrated in to the patient’s brain, express a broad array of natural and potent protective agents providing preservation and protection against injury, toxins, and neurodegenerative diseases such as Parkinson’s. The study was published in The FASEB Journal.

Here’s the study.

DNA Double Take

A genome is a genome is a genome? Not always. Turns out that many people can have more than one genome in their body at once. Now that sequencing has become cheaper to do, all sorts of interesting data like this is popping up. From the NYT:

Scientists are discovering that — to a surprising degree — we contain genetic multitudes. Not long ago, researchers had thought it was rare for the cells in a single healthy person to differ genetically in a significant way. But scientists are finding that it’s quite common for an individual to have multiple genomes. Some people, for example, have groups of cells with mutations that are not found in the rest of the body. Some have genomes that came from other people.

Researchers Grow Human ‘Mini Brains’

This advance by Austria’s Dr. Knoblich has big implications for studying brain disease. If researchers can study human brain cells in the lab instead of using animal models, the door will open to better, more effective, therapies. From the WSJ:

Researchers have used stem cells to grow pea-sized structures that resemble the developing human brain, an advance that offers a way to model brain maladies that are otherwise hard to study.

The human brain is one of the most elaborate natural structures known to science. These new lab-grown “mini brains” are imperfect, and a long way off from matching the real thing.

The advance is expected to allow researchers to investigate human brain disease in a lab—something that currently is a big challenge. Brain disorders such as Alzheimer’s typically are studied in rats, mice and other animals, but these are inadequate proxies mainly because the human brain is much more complex.

By contrast, the new approach should enable scientists to study neurological disorders by examining brain tissue derived from actual patients.