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Gene Edits for Treatment of Disease

Most scientists have applied the CRISPR/Cas system to specific tissues or to stem cells. Bedford Research scientists are applying the technology to edit B2M gene sequences in unfertilized eggs which are subsequently activated for stem cell derivation. Last year a Portland Oregon research team reported their efforts to repair a mutation in the gene MYBPC3 known to be associated with acute heart failure in young men.

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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…

Gene Edits for Research

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 and Mr. Little’s Fancy Mice, bred for coat color, formed the basis of the Jackson Laboratory’s inbred mice to study genetic 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…

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%…

Naturally Occurring Gene Edits

No two individuals have exactly the same gene sequences because multiple sequences code for the same amino acid. This is the basis for DNA tests to prove paternity or predict ancestry. Most of the gene variations do not change the proteins they code for, but some do, such as genes for eye and hair color and height.

What is Gene-Editing?

Is gene editing human embryos a positive scientific breakthrough for human health, or misuse of a powerful research tool? Over the next six months, we will outline the basic biology behind gene editing, followed by a description of the process in general and in human embryos, specifically.