9 GREAT TALKS ON CIRCADIAN RHYTHMS & EARLY DEVELOPMENT

Each of these talks was filmed live at our recent Activated Egg Symposium. Egg and stem cell researchers from around the world gathered to discuss the role of circadian rhythms in early human development and stem cell differentiation during this unique, one-day workshop.

Gene Editing Human Embryos: Who Should Decide?

Dinner Speaker: Arthur Applbaum, PhD
Arthur Isak Applbaum, PhD, is Professor of Ethics and Public Policy and Director of Graduate Fellowships in the Harvard University Center for Ethics and the Professions.

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Bedford Research Foundation Clinical Laboratory Testing for COVID19

Please DONATE to support this urgently needed FREE testing program.

**FOR IMMEDIATE RELEASE**

BEDFORD RESEARCH FOUNDATION CLINICAL LABORATORY WILL BE TESTING FOR COVID19.

As part of its mission to support treatment of currently incurable diseases in communities, the Bedford Research Foundation clinical laboratory, located in Massachusetts, will begin offering a highly sensitive highly specific test for SARS-2 (severe acute respiratory syndrome-2), the new coronavirus responsible for Corona Virus Induced Disease, 2019 (COVID19).

Bedford Research scientists are currently finishing the approval process for the Food and Drug Administration (FDA), and plan on ramping up the test immediately upon completed review process. The BRF test is based on thirty years of experience as a CLIA (Clinical Laboratory Improvement Amendments) – licensed FDA-registered lab dedicated to detecting HIV and Hepatitis C in a variety of clinical specimens.

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Bedford Research Foundation 2019 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 23! Dr. Kiessling outlines her vision for the upcoming year as well. Thank you for your support.


Bedford Research Foundation is TWENTY THREE

Founded in 1996 to conduct research that cannot be funded by the National Institutes of Health, Bedford Research scientists have achieved ground-breaking milestones!

See our Timeline of Milestones!

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DNA render

Gene Edits for Enhancement

The fifth installment of our blog series about gene editing focuses on gene edits and editing for research purposes. We hope you that you find it informative – please Contact Us with any comments! View the other posts in this series!

Earlier this year, a Chinese scientist reported the birth of twin girls whose genomes had been modified to silence the CCR5 gene.

Human genome: All of the genetic information needed for the embryonic development and adult function of a human being.

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Gene Edits for Treatment of Disease

The fourth installment of our blog series about gene editing focuses on gene edits and editing for research purposes. We hope you that you find it informative – please Contact Us with any comments! View the other posts in this series!

Gene: A specific sequence of A, C, G, T units that instruct the sequence of amino acids that comprise a specific protein. Humans have 20- to 25 thousand genes

As part of our ongoing blog series about gene editing techniques and uses, the Bedford Research Foundation presents:

Gene Edits for Treatment of Disease

Most scientists have applied the CRISPR/Cas system to specific tissues or to stem cells. For example, it is theoretically possible to repair the X-chromosome mutations in liver cells so normal blood clotting factors can be produced by the liver.

CRISPR/Cas: “Clustered Regularly Interspaced Short Palindromic Repeats” is a term that describes DNA sequences in the viruses that infect bacteria. The immune system of bacteria includes a family of proteins (CRISPR-associated, Cas) that recognize CRISPR sequences and degrades them. The enzyme, Cas, needs to bind to a specific RNA sequence of 120 units, which can be synthesized synthetically, in order to degrade the DNA. These two components also function well in cell types other than bacteria, and so have become a useful tool for cutting DNA, resulting in either small deletions, or successful insertions of new synthetic DNAs. Both outcomes create an edited (mutated) gene.

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Bedford Research Foundation Fact Sheet

OUR MISSION

Bedford Research Foundation is a Massachusetts 501(c)(3) public charity and biomedical institute conducting stem cell and related research for diseases and conditions that are currently considered incurable.

WHAT WE DO

BRF conducts research in three principal areas: stem cells, prostate disease and HIV/AIDS.

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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 you donate 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 of $450,000 in 2018.

Gene Edits for Research

The third installment of our blog series about gene editing focuses on gene edits and editing for research purposes. Hope you that you find it informative – please Contact Us with any comments! View the other posts in this series!

Gene edit: A modification of a specific sequence of A, C, G, T units that instruct the sequence of amino acids that comprise a specific protein. The edit may or may not alter the amino acid sequence and the protein.

Early gene editing experiments were accomplished by mating individuals with different traits. Two well known examples are Mendel’s famous red peas crossed to white peas to yield pink peas (Mendel experiments summarized in this short animation: https://youtu.be/Mehz7tCxjSE), and Mr. Little’s Fancy Mice, popular in the early 1900’s, bred for coat color, formed the basis of the Jackson Laboratory’s inbred mice to study genetic diseases.

Nobel Laureate Mario Capecchi systematically studied the function of mouse genes by mutating them into silence, so called “knock-out” mice (he also spoke at the Foundation’s annual Activated Egg Symposium, in a talk titled “Gene Targeting Into the 21st Century: Mouse Models of Human Disease From Cancer to Neuropsychiatric Disorders”). This was accomplished by flooding cultures of mouse embryonic stem cells with strands of synthetic DNA that could replace the normal gene with an edited copy during DNA replication. The edited gene sequence was designed to not guide the synthesis of the normal protein. Such gene edited cells were combined with early mouse embryos, ultimately becoming part of the tissues of the mouse, including occasionally sperm and eggs. Males with gene edited sperm were mated to females with gene edited eggs to produce offspring containing two copies of the edited, non-functioning genes. Although laborious and time-consuming, this approach has yielded highly valuable information about the normal functions of thousands of genes.

In the past 20 years, other less time consuming methods of silencing genes, or increasing their expression, have been developed, all with the goal of understanding their function in health and disease.

In 2013, the most recent method for gene editing was popularized by scientists at Stanford and MIT. It is an adaptation of a naturally occurring defense mechanism that bacteria have against the viruses that invade them. Termed CRISPR/Cas, it is a complex between a protein that can cut DNA strands and a synthetic single-stranded RNA with a sequence of A, C, G, U that matches the gene being targeted (short video explanation of CRISPR here: https://youtu.be/duKV1lNiqQw). The simplicity and specificity of the system have rapidly led to a wide variety of applications among scientists world-wide.

CRISPR/Cas: “Clustered Regularly Interspaced Short Palindromic Repeats” is a term that describes DNA sequences in the viruses that infect bacteria. The immune system of bacteria includes a family of proteins (CRISPR-associated, Cas) that recognize CRISPR sequences and degrades them. The enzyme, Cas, needs to bind to a specific RNA sequence of 120 units, which can be synthesized synthetically, in order to degrade the DNA. These two components also function well in cell types other than bacteria, and so have become a useful tool for cutting DNA, resulting in either small deletions, or successful insertions of new synthetic DNAs. Both outcomes create an edited (mutated) gene. 

Such targeted DNA cuts can edit the gene sequences so they no longer code for a functioning protein, analogous to the natural CCR5 mutation, or opening the DNA strands can allow the incorporation of synthetic DNA sequences into the cut site. This raises the exciting possibility of being able to repair defective human genes. We’ll see you next month, when we’ll discuss how these research gene editing techniques may be used in the potential treatment for diseases.

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.

From the Director

The derivation of gene edited, universal, HIV-resistent 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