Bedford Research Foundation 2017 Newsletter
Read about all of the progress and the research that has occurred at the Foundation over the course of the past year, and a retrospective on the past 21! Dr. Kiessling outlines her vision for the upcoming year as well. Thank you for your support.
Bedford Research Foundation is TWENTY ONE
See our Timeline of Milestones!
BRF Research News
Our goal for 2017 was to improve the efficiency of a new technology, “gene editing” by CRISPR, that can precisely edit genes in eggs activated to become stem cells. BRF scientists accomplished this goal in a mouse model by developing new methods that improve the efficiency of CRISPR gene editing in mouse eggs from 10% to approximately 75%, with the added success of deriving stem cells from more than 50% of the gene edited, activated eggs.
Two genes were simultaneously targeted for editing:
(1) Just as Type “O” blood can be given to almost everyone, a “universal” stem cell could be missing the gene, B2M, responsible for the proteins on stem cells that cause immune rejection following transplantation. Such a “universal” stem cell could be transplanted into many individuals without leading to immune rejection. This is an essential step to the derivation of “off-theshelf” stem cells for everybody.
The 2017 mouse egg stem cell experiments by BRF scientists derived mouse stem cells missing B2M. This paves the way to translate the research to the derivation of universal stem cells from human eggs. Like blood banks, universal stem cell banks would be available in hospitals for acute treatments, such as heart attack, stroke and spinal cord injury.
(2) CRISPR gene editing can also mimic the natural mutation in 1% of humans that renders individuals resistant to infection by HIV, the virus that causes AIDS. The recent success in mouse eggs to eliminate the HIV receptor, CCR5, paves the way to deriving a library of universal human stem cells also resistant to HIV infection.
IF those cells can be developed into bone marrow stem cells, and IF those bone marrow stem cells will function normally, they could be utilized as a powerful treatment, perhaps a cure, for HIV disease.
New Research Program a Success in Mouse Stem Cells
Dr. Joel Lawitts microinjects CRISPR/Cas “gene editing” enzymes into mouse eggs to neutralize two genes at once: (1) the gene that leads to tissue rejection, and (2) the gene that allows HIV infection of cells. These are the first steps in generating off-the-shelf stem cells for everybody that are also resistant to HIV infection.
“Dr. Kiessling and her staff have shown their determination to tackle some of the most difficult health problems of our time.”
– Representative Ken Gordon
Along with continuing patient recruitment into the prostate cancer screening project, BRF Scientists have developed methods for including specimens submitted to the laboratory for other types of testing. The goal of the project is to develop semen screening tests that improve diagnosis and staging of prostate cancer as well as reflect overall male health.
Alex Hauser graduated from UMass Lowell with a B.S. in Biology in 2017 and joined the BRF staff full time. Alex is focused on the SPAR program and also has an interest in gene editing technology and analysis of cell lines.
Dr. Bronte Stone holds a PhD in Medicine and has directed the Laboratory of California Fertility Partners with over 30 years experience in human eggs and embryo development. Dr. Stone joins our scientific advisory board as we enter the human egg phase of our research.
The Massachusetts Life Science Center sponsors internships in companies engaged in life science development in the Commonwealth. Taylor Mellen will graduate from UMass Lowell in 2018 with a B.S. Degree in Biology. We’ve enjoyed having Taylor with us working in the andrology lab services.
From the Director
The derivation of gene edited, universal, HIV-resistant human stem cells from unfertilized eggs will not be without controversy. Fortunately, we have meritorious individuals serving as our Ethics Advisory Board, our Human Subjects Committee and our Stem Cell Research Oversight Committee. Their guidance has helped us forge ahead into areas of stem cell development that larger institutions have shied away from because the work cannot be funded by our federal government. The “Dickey-Wicker Amendment” to the budget of the National Institutes of Health has been renewed annually and prohibits funds to be used for studies of unfertilized human eggs. We have for years believed unfertilized eggs (“parthenotes”) will be a broadly applicable source of “universal” human stem cells for everybody. Since human egg research MUST be privately funded, progress depends entirely on private donations. BRF is uniquely positioned to push this exciting field forward, and we need everyone’s support!
Ann A Kiessling, PhD
Director, Bedford Research Foundation
Who is Bedford Research Foundation?
Philanthropy Is The Key To Continued Progress
The average cost of each experiment is $90,000. Because much of our overhead is covered by fee-for-service laboratory tests, 92% of every dollar donated goes directly toward these experiments. This innovative funding model allows Bedford Research scientists greater flexibility to move quickly in promising new research directions.
Continued progress requires meeting our annual funding goal.