Scientists Correct Mutated Gene that Causes Sickle Cell Disease in Stem Cells


For the first time, scientists were able to correct the genetic mutation that causes sickle cell disease in stem cells.

In a collaborative effort, researchers at UC Berkeley, UC San Francisco Benioff Children’s Hospital Oakland Research Institute (CHORI), and the University of Utah School of Medicine fixed the mutation in modified stem cells from patients with the condition using a CRISPR/Cas9 gene editing approach.

The study, “Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells,” was published in the journal Science Translational Medicine.

The scientists hope to re-infuse patients with the modified stem cells and alleviate disease symptoms.

“We’re very excited about the promise of this technology,” Jacob Corn, senior author on the study and scientific director of the Innovative Genomics Initiative at UC Berkeley, said in a news release. “There is still a lot of work to be done before this approach might be used in the clinic, but we’re hopeful that it will pave the way for new kinds of treatment for patients with sickle cell disease.”

The researchers observed in mice tests that after transplants, the modified stem cells stuck around for about four months, an important target of the long-lasting potential of any therapy.

“This is an important advance because for the first time we show a level of correction in stem cells that should be sufficient for a clinical benefit in persons with sickle cell anemia,” said Mark Walters, a pediatric hematologist and oncologist and director of UCSF Benioff Oakland’s Blood and Marrow Transplantation Program and co-author of the study.

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These companies search for a cure to aging– and their discoveries are amazing


The ideas surrounding life enhancement are not new—in fact, records show an interest in the mysteries surrounding human life for centuries.

Mary Shelley’s Frankenstein explores the idea of creating a life, while Doctor Who achieves life extension through regeneration. Wolverine’s mutations let him heal away his problems, and Captain America froze himself into the 21st century. Just look at almost any Star Trek episode and you’ll see how fascinated people are with the idea of extending life.

These ideas are starting to extend beyond science fiction. What was once seen as fiction is, in fact, highly relevant in today’s scientific community. Life extension research lives in academia at the moment, but it’s also graining traction in nonprofit foundations and national organizations.

This scientific field aims not only to discover the solutions to life’s unanswered aging questions, but also allow humanity to “live long and prosper.”

Why bother with this research?

The “holy grail” of the life extension industry is the cure to aging (obviously) and its discovery would change the course of human history forever.

However, when looking at life extension from the viewpoint of the Average Joe, there are many very real, personal, and emotional reasons which can be tied to the desire for those extra years.

“Seeing friends and family age can be difficult to go through,” said Dr. Chris Barton, assistant professor of biology at Belmont University in Nashville, TN. “As a result, I think that we are becoming more conscious of the aging process and more intentional about trying to find ways to delay it.”

When did life extension research really begin?


calico logo

Hopefully Calico doesn’t go the way of Google Glass. (Credit: Calico)

Interest in life extension has existed for decades– one of the largest booms in life extension research began in the 1990s. In 1992, The American Academy of Anti-Aging Medicine was established to explore the mysteries behind our bodies’ aging process.

From then the new millennium began, and with it came companies such as the Methuselah Foundation, co-founded by Dave Gobel and Dr. Aubrey de Grey in 2003, and through its leadership came the “Strategies for

Engineered Negligible Senescence,” or SENS Research Foundation, founded in 2009. In 2013, Google announced its new company Calico, who under the leadership of Arthur D. Levinson would focus on human health in relation to aging and its associated diseases.

“Nothing breeds success better than success,” Dr. Barton explained when reviewing the recent boom in anti-aging research out of these foundations. “While many of these advancements are in basic science research, it is really this foundational understanding of aging that has allowed us to detect and treat numerous aging-related diseases.

“If you look at the life expectancy data from 1960 to today, people are clearly living longer”—life expectancy in the United States alone jumped from age 70 in 1960 to age 79 in 2014, according to The World Bank. “We are currently more effective in treating conditions such as cardiovascular disease, cancer, and other aging-related diseases than we were 30 years ago. I think the recent success we’ve had in these areas is developing an excitement for aging research that can perhaps generate discoveries and technologies that may even further extend our life expectancy,” he continued.

The different areas of life extension research

These companies challenge current researchers and scientists to study the mysteries surrounding aging. The Methuselah Foundation focuses on Organovo and the ability to 3D print functional human tissues with hopes of creating functioning organs, while the SENS Research Foundation focuses on rejuvenation biotechnologies with new therapies which target and repair molecular damage responsible for the body’s aging.

With a Ph. D. in Biochemistry from Vanderbilt University and specializations in physiology, cell biology, and molecular genetics, Dr. Barton was able to provide insight into one of the many areas of research currently being studied among those in the field of life extension and anti-aging.

“Perhaps one of the most popular views behind the aging process is the ‘stem cell theory of aging,’ which states that as we age, our stem cells aren’t able to continue dividing to replenish the cells that are being lost in our tissues and organs,” Dr. Barton explained, believing this to be an area of research holding great promise.

“In addition, every time a cell divides there is the potential for it to accumulate some type of damage to its DNA. Given that stem cells must divide over an entire lifetime, they tend to accumulate quite a bit of damage. It is really the inability of our stem cells to continue growing indefinitely that many believe is the root of the aging process. Without a healthy pool of stem cells, tissues and organs are no longer able to maintain themselves in a way that supports life.”


human cell cross section

Learning how to reverse cell damage could be the key to reversing aging. (Credit: Thinkstock)

The hope for researchers is to promote the field and provide the world with a hope for advancement and, one day, a solution.

“Scientific progress, particularly in academia, is most often hindered by the decreases in government funding,” Dr. Barton said. “When large organizations such as these are willing to contribute funds or resources in order to advance research on a specific topic, I think they immediately become relevant to the larger research community.”

And, in the case of anti-aging and life extension research communities, the relevancy of their research extends much further than that in everyday culture, aging treatments, diseases associated with aging, life expectancy, and the overall quality of life every single person will one day encounter with age.

So why haven’t we found the solution to aging yet?


Irish scientists discover way to ‘print’ new bones to help those with deformities and catastrophic injuries


Trinity College
Trinity College


Irish scientists have developed a revolutionary new process which allows them to make human bones using 3D printing.

The new process could eliminate the need for bone grafts and could even make new joints to replace hips and knees and offers hope to those with large and complex bone defects or who have suffered catastrophic injuries.


In the future the process will allow bones to be repaired or even replaced cutting out the need for bone grafts.  Scientists at the Science Foundation Ireland-funded AMBER materials science centre at Dublin’s Trinity College have developed the new method of making bone material.  This is done using 3D bioprinting technology to construct cartilage templates in the shape of the missing bones.

When this is done the made up bone and stem cells is implanted under the skin, where it matures in time into fully functioning replacement bone with its own blood vessels.

The team, headed by Professor Daniel Kelly hope that in the future the development could lead to numerous applications in areas like head, jaw and spinal surgery.  READ MORE

Making memories stronger and more precise during aging

Young neurons (pink), responsible for encoding new memories, must compete with mature neurons (green) to survive and integrate into the hippocampal circuit. Photo courtesy of Kathleen McAvoy, Sahay Lab.
Young neurons (pink), responsible for encoding new memories, must compete with mature neurons (green) to survive and integrate into the hippocampal circuit. Photo courtesy of Kathleen McAvoy, Sahay Lab.

HSCI researchers identify new mechanisms by which new neurons sharpen memories

By Hannah L. Robbins, HSCI Communications

When it comes to the billions of neurons in your brain, what you see at birth is what get — except in the hippocampus. Buried deep underneath the folds of the cerebral cortex, neural stem cells in thehippocampus continue to generate new neurons, inciting a struggle between new and old as the new attempts to gain a foothold in the memory-forming center of the brain.

In a study published online today in Neuron, Harvard Stem Cell Institute (HSCI) researchers atMassachusetts General Hospital and the Broad Institute of MIT and Harvard in collaboration with an international team of scientists found they could bias the competition in favor of the newly generated neurons.

“The hippocampus allows us to form new memories of ‘what, when and where’ that help us navigate our lives,” said HSCI Principal Faculty member and the study’s corresponding author, Amar Sahay, PhD, “and neurogenesis—the generation of new neurons from stem cells—is critical for keeping similar memories separate.”

As the human brain matures, the connections between older neurons become stronger, more numerous, and more intertwined, making integration for the newly formed neurons more difficult. Neural stem cells become less productive, leading to a decline in neurogenesis. With fewer new neurons to help sort memories, the aging brain can become less efficient at keeping separate and faithfully retrieving memories.

The research team selectively overexpressed a transcription factor, Klf9, only in older neurons in mice, which eliminated more than one-fifth of their dendritic spines, increased the number of new neurons that integrated into the hippocampus circuitry by two-fold, and activated neural stem cells.   READ MORE

Michael Sefton to receive Lifetime Achievement Award from the Tissue Engineering & Regenerative Medicine International Society

University Professor Michael Sefton (IBBME, ChemE), University of Toronto biomedical engineering University Professor Michael Sefton (IBBME, ChemE) has been awarded the Lifetime Achievement Award from the Tissue Engineering & Regenerative Medicine International Society. (Credit: Neil Ta)
University Professor Michael Sefton (IBBME, ChemE), University of Toronto biomedical engineering University Professor Michael Sefton (IBBME, ChemE) has been awarded the Lifetime Achievement Award from the Tissue Engineering & Regenerative Medicine International Society. (Credit: Neil Ta)

University of Toronto biomedical engineering University Professor Michael Sefton (IBBME, ChemE) has been named this year’s recipient of the Lifetime Achievement Award from the Tissue Engineering & Regenerative Medicine International Society (TERMIS). The award, issued by the organization’s Americas chapter, recognizes his immense contributions to the fields of tissue engineering and regenerative medicine.

Sefton joins an elite list of renowned recipients, including MIT professor of chemical engineering Robert Langer and founding director of the University of Pittsburgh’s McGowan Institute for Regenerative Medicine, Alan Russell.

Sefton has made significant contributions to research advances in biomaterials, biomedical engineering and regenerative medicine. He was one of the first to combine living cells with polymers, effectively launching the field now now called tissue engineering. Recently, his lab has created biomaterials that actively promote the growth of blood vessels — such materials accelerate wound healing and support the development of lab-grown tissues.  READ MORE

Functional human tissue-engineered liver generated from stem, progenitor cells


Biodegradable scaffold (left) and human tissue-engineered liver (right). Credit: The Saban Research Institute at Children's Hospital Los Angeles
Biodegradable scaffold (left) and human tissue-engineered liver (right).
Credit: The Saban Research Institute at Children’s Hospital Los Angeles

A research team led by investigators at The Saban Research Institute of Children’s Hospital Los Angeles has generated functional human and mouse tissue-engineered liver from adult stem and progenitor cells. Tissue-engineered Liver (TELi) was found to contain normal structural components such as hepatocytes, bile ducts and blood vessels. The study has been published online in the journal Stem Cells Translational Medicine.

Liver disease affects pediatric and adult patients, impacting one in ten people in the United States. Liver transplantation is the only effective treatment for end-stage liver disease, but scarcity of available organs and the need for lifelong immunosuppressive medication make this treatment challenging.

Alternate approaches that have been investigated include significant limitations. For example, conventional liver cell transplantation requires scarce donor liver and a perfusion protocol that wastes many cells. This type of cell transplant typically lasts less than one year, with most patients ultimately requiring a liver transplant. Human-induced pluripotent stem (iPS) cells are another possibility but, so far, iPS cells have remained immature rather than developing into functional and proliferative liver cells, called hepatocytes. There continues to be a need for a durable treatment, particularly one that could eliminate the need for immunosuppression.  CONTINUE READING…

Methuselah Foundation Fellowship Award Winner Tackles Research in Macular Degeneration 

Jennifer Rosa small

Typically, a fellowship and participation in a research study to cure a major disease would occur years after completing undergrad, possibly even after earning a PhD. But Jennifer DeRosa is not a typical student.

As early as high school, DeRosa was already in the lab, conducting research in plant biotechnology at the College of Environmental Science and Forestry (SUNY-ESF) before graduating valedictorian from Skaneateles High School. As a freshman student at Onondaga Community College, she continued to develop skills in molecular biology, analytical chemistry, and cell biology. She logged over 1,600 hours in academic and industry laboratories while maintaining a perfect 4.0 GPA, completing her associate’s degree in Math and Science in only one year.

Although she had planned to continue to a bachelor’s program, DeRosa elected to defer enrollment after being offered a Methuselah Foundation research fellowship. “The fellowship provides distinguished students a year-long stipend to work in any laboratory of their choosing that conducts work on age-associated diseases,” said Methuselah Foundation CEO David Gobel. “We are very pleased that she chose to complete her fellowship at Ichor Therapeutics, where she has been working as a paid intern. Methuselah Foundation has a high degree of confidence in the quality and scope of work being conducted there.”

Her enthusiasm for her work has caught the attention of everyone who works with her. “Jennifer [DeRosa] has distinguished herself at every level since beginning as an intern in January,” stated Ichor’s Quality Assurance Director Scott Campbell. “We are delighted about her decision to stay on and help us drive our age-related macular degeneration program into the next stage of development, including adopting of stringent GMP and GLP regulatory requirements.”

DeRosa is excited about the research that Ichor Therapeutics is currently engaged in, as well as the opportunities to learn in areas beyond the science itself. She said, “I chose to intern at Ichor because as a startup, I knew it would allow me to explore entrepreneurship and take on a greater role than I otherwise could at a large company. Between being able to participate in board meetings, discuss legal and translational strategy with Ichor’s counsel and advisory teams, and meeting the company’s investors to better understand their expectations – Let’s just say it was a simple decision for me to remain here.”

DeRosa’s previous research at Ichor substantially and directly contributed to the company successfully raising $600,000 for its macular degeneration program earlier this summer. DeRosa was a listed author on both the research proposal and business plan, and is also listed on two pending grant applications.

Kelsey Moody, CEO at Ichor Therapeutics, noted, “The most difficult part of having her here is finding sufficient challenges. She has earned complete autonomy since her arrival. Beyond her expansive laboratory skills, she has designed her own studies, written proposals for grants, and led a small team to develop product leads for the macular-degeneration program.”

When her fellowship draws to a close, DeRosa intends to pursue a bachelor’s degree or matriculate directly into a graduate program. However, she plans to remain opportunistic. “The pace, progress, and potential impact of Ichor’s macular degeneration program is addicting. The company’s main focus now is to prepare for series A, after which, who knows what opportunities may present themselves.” 

Vascular Tissue Challenge Introduction Webinar


Event Description

The Vascular Tissue Challenge is a $500,000 prize purse for the creation ofthick, human vascularized organ tissue in an in-vitro environment that maintains metabolic functionality similar to in vivo native cells throughout a 30-day trial period. The Methuselah Foundation’s New Organ Alliance and NASA’s Centennial Challenges Program have partnered to create this challenge with the goal of advancing research on human physiology, fundamental space biology, and medicine taking place both on the Earth and the ISS National Laboratory.

Additionally, the Center for Advancement of Science in Space (CASIS) will be providing an additional “Innovations in Space” Award covering $200,000 in hardware costs and the launch costs to send one team’s vascular tissue experiment to the International Space Station that could further their research in the field.

This introductory webinar is for anyone interested in potentially competing for the Vascular Tissue Challenge. In the webinar we will discuss the following items:

  1. Introduction to the Methuselah Foundation, New Organ Alliance, and NASA Centennial Challenges.
  2. Goals of the Vascular Tissue Challenge.
  3. Rules & evaluation criteria review.
  4. Innovations in Space Awardresearch opportunity using microgravity environment onboard the ISS.
  5. Processes and Procedures to compete.
  6. Question & Answer session.

Registration is free, but is required. The web address for the webinar will be emailed to webinar registrants prior to the event.


Episode 007 – Control Alt Delete Cancer Research into longevity, human health, ageing, New Organ Research including bioprinting liver, kidney, and other medical pursuits


Control Alt Del

Hello and welcome to Episode 7!  On this episode, we’ll talk with Dr. Haroldo Silva and David Halvorsen of the SENS Research Foundation.  They’ve launched a new crowdfunding campaign designed to attack and stop cancer using a new approach.  You’ll hear what that approach is, why they think it has a good chance of success, and you can help in the fight.

If you’d like to comment on the show, have a question or want to reach us, write

To learn how you can help the Methuselah Foundation continue its mission to extend heathy human life, CLICK HERE