Tag Archives: science

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

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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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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

BREAKING: Methuselah Partnering with NASA

June 13, 2016

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NASA Challenge Aims to Grow Human Tissue to Aid in Deep Space Exploration

NASA, in partnership with the nonprofit Methuselah Foundation’s New Organ Alliance, is seeking ways to advance the field of bioengineering through a new prize competition.

The Vascular Tissue Challenge offers a $500,000 prize to be divided among the first three teams that successfully create thick, metabolically-functional human vascularized organ tissue in a controlled laboratory environment.

“The humans who will be our deep space pioneers are our most important resource on the Journey to Mars and beyond,” said Steve Jurczyk, associate administrator for NASA’s Space Technology Mission Directorate in Washington. “The outcome of this challenge has the potential to revolutionize healthcare on Earth, and could become part of an important set of tools used to minimize the negative effects of deep space on our future explorers.”

Related cells that are joined together are collectively referred to as tissue, and these cells work together as organs to accomplish specific functions in the human body. Blood vessels around the cells vascularize, providing nutrients to the tissue to keep it healthy. The vascularized, thick-tissue models resulting from this challenge will function as organ analogs, or models, that can be used to study deep space environmental effects, such as radiation, and to develop strategies to minimize the damage to healthy cells. 

Studying these effects will help create ways to mitigate negative effects of space travel on humans during long duration, deep space missions. On Earth, the vascularized tissue could be used in pharmaceutical testing or disease modeling. The challenge also could accelerate new research and development in the field of organ transplants.

When the Wright Brothers discovered how to control aircraft during flight for aviation in the early 1900s, there was an explosion of progress after this key barrier was removed”, said Dave Gobel, chief executive officer of the Methuselah Foundation. “In the same way, once the ‘vascularization limit’ is solved, via the NASA Vascular Tissue Challenge, there inevitably will be an historic advance in progress and commercialization of tissue engineering applications to everyone’s benefit.”

Competitors must produce vascularized tissue that is more than .39 inches (1 centimeter) in thickness and maintains more than 85 percent survival of the required cells throughout a 30-day trial period. Teams must demonstrate three successful trials with at least a 75 percent success rate to win an award. In addition to the laboratory trials, teams also must submit a proposal that details how they would further advance some aspect of their research through a microgravity experiment that could be conducted in the U.S. National Laboratory on the International Space Station.

The new challenge was announced as part of White House Organ Summit, which highlighted efforts to improve outcomes for individuals waiting for organ transplants and support for living donors. In a related initiative, the Center for the Advancement of Science in Space (CASIS), which manages the International Space Station U.S. National Laboratory, announced a follow-on prize competition in partnership with the New Organ Alliance and the Methuselah Foundation that will provide researchers the opportunity to conduct research in microgravity conditions. CASIS will provide one team up to $200,000 in flight integration support costs, along with transportation to the ISS National Laboratory, support on station and return of experimental samples to Earth. CASIS also announced the winners of the $1 million 3-D Microphysiological Systems for Organs-On-Chips Grand Challenge.

The Vascular Tissue Challenge prize purse is provided by NASA’s Centennial Challenges Program, part of NASA’s Space Technology Mission Directorate. Centennial Challenges, managed by the agency’s Marshall Space Flight Center in Huntsville, Alabama, is NASA’s citizen inventor prize program that invites the nation to help advance the technologies that will enable us to go to Mars and beyond, as well as improve life on Earth. The New Organ Alliance, which is administering the competition on behalf of NASA, is a nonprofit organization focused on regenerative medicine research and development to benefit human disease research and tissue engineering.

For information about the Methuselah Foundation’s New Organ Alliance, official challenge documents, rules and schedule of events, visit:

https://neworgan.org/vtc-prize.php

For more information about the Vascular Tissue Challenge, visit:

http://www.nasa.gov/vtchallenge