Bedford Research Foundation team and Rep. Gordon responding to need for COVID testing

As soon as she saw the community spread of the COVID-19 virus in Washington State, Bedford Research Foundation’s (“BRF”) Clinical Laboratory Director Dr. Ann Kiessling sprang into action. Just two weeks later, with the help of Rep. Ken Gordon (D-Bedford), BRF is processing hundreds of tests each week for patients exhibiting symptoms that could be caused by the COVID-19 virus, or were directly exposed to it.

“We had the resources to help, and we felt it was our responsibility to pitch in,” said Dr. Kiessling

With 30 years of experience as a Clinical Laboratory Improvement Amendments (“CLIA”)-licensed FDA-registered lab dedicated to detecting HIV and Hepatitis C, Dr. Kiessling set about to convert her lab to testing for the Severe Acute Respiratory Syndrome-2 (“SARS-2”) coronavirus responsible for COVID-19. The CLIA certification allowed BRF a streamlined process for testing under Food and Drug Administration (FDA) guidelines, and because of that, Dr. Kiessling and her staff were able to begin testing samples in a matter of days.

The next problem was obtaining access to those samples. Dr. Kiessling called the Center For Disease Control (“CDC”) to tell it she could help with Massachusetts’ need. The CDC was excited to have a new source for testing, but had not identified a population center she could serve. She called local hospitals, and while the response was positive, she was put on hold. The Governor’s COVID19 emergency center was organizing the response.

That’s when Dr. Kiessling put in a call to Rep. Gordon, explaining that as a local alternative with faster turn-around than larger, more remote labs, BRF will supplement the CDC-based resources. She was willing to provide testing at cost. Rep. Gordon immediately followed up with a call to Health and Human Services Secretary Marylou Sutters, who doubles as chief of the COVID-19 crisis center. A few days later the connection was made. Dr. Kiessling got a call back from Emerson, telling her that the Commonwealth told them to send samples to her. Not long after that, BRF began receiving samples from Sturdy Memorial Hospital in Attleborough.

BRF is currently testing hundreds of samples each week. It can provide results within 48 hours.

“We had the resources to help, and we felt it was our responsibility to pitch in,” said Dr. Kiessling. “We have to be prepared for the number of people who display symptoms or have been in contact with those testing positive for the virus to increase. As a matter of public health, we need to know where the virus has spread, and we’re happy to do our part.”

Similarly, Rep. Gordon was proud that a lab in his district was willing to be part of the solution.

“Ann came to me as this outbreak was just beginning. She’s a respected and conscientious HIV researcher, and she explained how she could adapt her experience to focus on his novel virus. There’s no doubt we need more testing in Massachusetts, and the greater access to reliable labs, especially Massachusetts labs, the better. I explained to Secretary Sudders that if Dr. Kiessling says she can do this, then she can. I’m glad the connection was quickly formed.”

Dr. Kiessling and her staff are not the only Bedford entries in the fight against the SARS-2 coronavirus. Julie Godon is an integral part of Abbott Labs’ marketing of a rapid Coronavirus test, that received FDA approval this week. This new test is expected to yield results in a matter of minutes.

BRF testing is available through a patient’s healthcare provider, all testing must be ordered through a clinic or Doctor’s office. The Bedford Research Foundation has set up a specialized response email for patients looking for information. Email: covid@bedfordresearch.org.

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.


Circadian Clock Mechanisms in Mammals and Their Relevance to Aging and Longevity

Circadian Keynote: Joseph Takahashi, PhD
Dr. Takahashi is Chair of the Department of Neuroscience at University of Texas Southwestern Medical Center.


Contributions of Oocyte Cytoplasm to Reprogramming and Development

Oocyte Keynote: Shoukhrat Mitalipov, PhD
Dr. Mitalipov is the Director of the Oregon Health & Sciences University Center for Embryonic Cell and Gene Therapy. He is also a Professor in the Division of Reproductive & Developmental Sciences of Oregon National Primate Research Center.


Oocyte Contributions to Embryogenesis: Mother’s Legacy

David Albertini, PhD
Dr. Albertini is a Senior Scientist at Bedford Research Foundation and Editor-in-Chief of the Journal of Assisted Reproduction and Genetics. Previously he served as a Professor of Molecular and Integrative Physiology at Kansas University Medical Center.


Post-transcriptional Regulation of Protein Expression by the Circadian Clock

Carla Green, PhD
Dr. Green is a professor in the Department of Neuroscience and a Distinguished Scholar in Neuroscience at the University of Texas Southwestern Medical Center.


Human / Mouse Chimeras for Disease Modeling

Rudolf Jaenisch, PhD
Dr. Jaenisch is Whitehead Institute Founding Member and National Medal of Science recipient.


First Evidence for Circadian Rhythms in Early Human Embryos

Ann A. Kiessling, PhD
Dr. Kiessling is Director of the Bedford Research Foundation and is retired Associate Professor of Surgery at Harvard Medical School.


The Circadian Clock as a Window into Neurodevelopment Disorders: Mechanisms and Opportunities

Jonathan Lipton, MD, PhD
Dr. Lipton is an Assistant Professor of Neurology, Harvard Medical School and Faculty at the Kirby Center, Boston Children’s Hospital.


Embryonic Circadian Clocks: Do They Control Anything Important?

David Whitmore, PhD
Dr. Whitmore is a Professor of Chronobiology in the Division of Biosciences at University College London.
Speakers in the front row from left: Arthur Applbaum, Jonathan Lipton, Carla Green, David Whitmore, Shoukhrat Mitalipov, Rudolph Jaenisch, Joseph Takahashi, and David Albertini.


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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.

New FDA guidance is designed for expedited review of test strategies for SARS-2 by CLIA-licensed labs. The BRF test will offer a local alternative with faster turn-around time for test results than larger commercial laboratories, such as Quest and LabCorp, and will supplement the CDC-based resources at the Mass Department of Public Health.

BRF scientists have developed a transport kit that inactivates the virus so specimens can be transported safety to the laboratory from clinical settings or patients’ homes for testing. Testing is available through a patients healthcare provider, all testing must be ordered through a clinic or Doctor’s office.

The Bedford Research Foundation has set up a specialized response email for patients looking for information, please email: covid@bedfordresearch.org.

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!

Stem Cells for Every Body

Unfertilized eggs can be activated artificially (parthenogenesis) to undergo cell multiplications similar to fertilized eggs, but do not give rise to offspring. At the time of activation, a protein responsible for tissue rejection can be silenced by gene editing.

“Universal donor” stem cells that are missing the major tissue rejection protein can then be derived from such edited parthenotes. Similar to Type O blood, such “universal” stem cells could be available “off-the-shelf” in emergency rooms for acute injuries, such as heart attack, stroke and spinal cord injury. This would be a major step forward in stem cell therapies for acute, as well as chronic conditions.


The Activated Egg Symposium 2019 brought together thought leaders in circadian rhythms, human egg parthenogenesis, human-animal chimeras for disease research and drug modeling, and the ethics of gene editing human embryos. It was an extraordinarily successful gathering with both speakers and attendees taking away new, important information. Unlike the large, commercial science meetings common today, registration was less than $100 for academics.

“Dr. Kiessling and her staff have shown their determination to tackle some of the most difficult health problems of our time.” – Representative Ken Gordon

Letter From The Director

We founded the Bedford Research Foundation as a unique, independent, not-for-profit research institution because of the prohibition on federal funding for our important work on unfertilized eggs (“parthenotes”). The result of our research will be a broadly applicable source of “universal” human stem cells for every body. We must forge ahead into areas of stem cell development that larger institutions shy away from because of the Dickey-Wicker Amendment that prohibits federally funded research on human eggs and the stem cells derived from them. Thanks to the guidance of the meritorious individuals serving on our Ethics Advisory Board, our Human Subjects Committee and our Stem Cell Research Oversight Committee, we are doing just that.

With our over 30 years of research experience in human egg biology and stem cell derivation, BRF scientists are uniquely qualified to push this exciting field forward. We were joined this year by Dr. David Albertini, a pioneer in egg biology, dedicated to understanding human eggs not only for stem cell derivation, but also to fill in critically important information gaps about human reproduction. Dr. Albertini’s research expertise will markedly accelerate our progress in successful stem cell derivation.

Our goals for 2020 include applying the research findings we have made in unfertilized mouse eggs to similar studies with unfertilized human eggs. The single copy of each chromosome in unfertilized eggs can be edited to eliminate the major protein on the surface of cells that causes tissues to be rejected following transplantation. Such “universal donor” stem cells can then be used as “off-the-shelf” treatments for acute conditions, such as heart attack, spinal cord injury and stroke, as well as chronic conditions, such as Parkinson’s Disease, diabetes, Lou Gehrig’s disease (ALS), Alzheimer’s Disease, and Huntington’s Disease. We won’t know the full therapeutic potential of human parthenote stem cells until the cells are actually derived.

Human egg research MUST be privately funded, please make a contribution to help us move this important work forward, we are most grateful for your support!

Sincerely,

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 of $450,000 in 2019.

Donate Today!

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.

The birth was reported to be one of a series of human embryo experiments designed to render the offspring resistant to infection by HIV and to prove the principal that gene editing was possible — and perhaps beneficial— in human embryos.

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

The work was not reported in a scientific format, so few scientists have had the opportunity to review the data in detail.

CCR5: A member of the C-C chemokine protein. receptor family that codes for the docking protein for the HIV virus on the surface of HIV target cells.

Several ethical concerns with this report, if true, have been raised. The gene editing was not performed to correct a known, serious medical issue in the embryos. It was performed to enhance resistance to HIV. A highly controversial idea.

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.

But a more practical problem with the work is the possibility of “off-target” gene edits. Much research has been devoted to discover, and eliminate, the random edits that may occur at other than the gene locations being specifically targeted by the CRISPR/Cas reagents.

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.

It is these potentially deleterious unintended consequences that must be addressed in order to protect the offspring produced.

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.

Bedford Research scientists are applying the technology to edit B2M gene sequences in unfertilized eggs which are subsequently activated for stem cell derivation.

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.

But more recently other scientists have applied CRISPR/Cas technology to human embryos. 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. The 30-member team created embryos with sperm from a man carrying the mutated gene in half of his sperm. (It is important to note that this experiment is not possible in Massachusetts because the stem cell bill (MGLc 111L) specifically prohibits the creation of embryos for research purposes only.)

At the time of fertilization of eggs with the mutant sperm, the Oregon scientists also injected the CRISPR/Cas agents designed to home to the gene mutation and insert “normal” DNA sequences. They reported the repair was successful in some embryos, but not all. Other research teams in New York and Australia replied to the report with their own interpretations of the results and all groups agreed much more work is needed to understand how to reliably edit genes in early human embryos.

We cannot put this genie back in the bottle, but with reasoned approaches, humans can optimize the benefits and mitigate the dangers posed by gene editing. Many organizations are using this technology to find solutions for diseases, such as:

The Bedford Research Foundation is using CRISPR/Cas to further HIV prevention work using early Parthenote egg cells – More about our work is posted here.

Thank you for reading, please come back again next month for our posting on “Gene Edits for Enhancement”.

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.

Stem Cells

Advances in stem cell biology have put within reach the possibility of cures for conditions such as Parkinson’s disease, spinal cord injury, heart disease and diabetes. What is needed is a reliable source of stem cells with the broad range of developmental potential (“pluripotent”) necessary for each cell type. Although controversial, the use of human eggs may be the most efficient way to generate pluripotent stem cells. Using mouse as a model system, BRF scientists are vigorously pursuing the possibility of generating pluripotent stem cells from unfertilized eggs, encouraged by the work of BRF Trustee Dr. Jose Cibelli who has successfully derived parthenotes from monkey eggs.

Background studies of early human embryos, conducted by BRF scientists in collaboration with medical researchers at the University of Athens, Greece, revealed a potentially important role for circadian rhythms in early embryos and stem cells. Studies are ongoing to discover what circadian genes are important to cell development, and to design culture conditions to support stem cell circadian rhythms.

In parallel, BRF scientists are taking advantage of a new technology, reported in 2013, to improve the efficiency of modifying stem cells for specific treatments, such as resistance to HIV infection, and development into essential cell types, such as nerves and immune cells.

Prostate Disease

Current research is expanding diagnostic capabilities of semen specimens to identify cancer and infections of the prostate. BRF scientists have developed the first comprehensive library of bacteria types detectable in semen specimens by state-of-the-art molecular biology techniques. This work is funded primarily through The Robert C. Eyre Research Fund. Dr. Eyre, one of the BRF’s medical researchers, is a leading urologic surgeon studying the etiology, diagnosis and treatment of diseases of the genitourinary tract, including infections and cancers of the prostate, kidney and bladder.

HIV/AIDS

In 1996, BRF began a Special Program of Assisted Reproduction (SPAR) to help men infected with HIV father children without transmitting the virus to mothers or babies. The program required BRF sponsorship because of federal statutes prohibiting funding from the National Institutes of Health (NIH) for research involving fertilized human eggs. The work was possible because BRF scientists have been studying HIV transmission since the beginning of the AIDS pandemic in the early 1980’s. As of April 2015, 230 HIV-free babies have been born through SPAR.

Current work is focussed on developing stem cells resistant to HIV infection. Since HIV infects blood cells through specific receptors on the surface of the cell, if that receptor were missing, the cell would not become infected. It has been known since the beginning of the AIDS pandemic that it would be possible to cure HIV infection by transplantation of bone marrow cells resistant to HIV, but it was not until 2009 that proof of this principle was obtained when an AIDS patient with cancer underwent a bone marrow transplant to cure his cancer with bone marrow cells that were naturally resistant to HIV because they were missing the HIV receptor (“CCR5”). Approximately 1.5% of humans are missing CCR5 on the surface of their cells.

To take advantage of this possible treatment/cure for HIV, BRF scientists are studying ways to efficiently “knock-out” the gene in stem cells. The most direct approach is to knock out the gene in the eggs before they develop into stem cells, because each succeeding cell will be genetically identical to the egg. Studies are ongoing.

WHO WE ARE

Ann A. Kiessling, PhD, Director of BRF, is an expert in HIV/AIDS and stem cell biology. Dr. Kiessling has published more than 100 scientific papers and is the author of Human Embryonic Stem Cells, the first textbook on the subject. Prior to devoting herself full time to the Foundation, she directed a lab at Harvard Medical School for over 25 years. BRF is guided by its Board of Trustees, made up of medical, legal and other experienced professionals, including Professor Jose Cibelli of Michigan State University, a pioneer in stem cell research, and Chairman Alan S. Geismer, Esq., of the law firm Sugarman, Rogers, Barshak & Cohen. The BRF Ethics Advisory Board is chaired by Arthur Applbaum, Professor of Ethics and Public Policy at Harvard’s Kennedy School of Government. Carol M. Warner, Matthews Distinguished Professor of Biology at Northeastern University, is chair of the BRF Science Committee.

BRIEF HISTORY

BRF began its work in 1996. Its initial project, the Special Program of Assisted Reproduction (SPAR), was created to facilitate healthy conception for couples with a male partner infected with HIV. The first SPAR baby was born healthy and infection-free in 1998. With the help of over 200 collaborating clinics nationwide, more than 230 healthy babies have been born through SPAR.

In addition to breakthroughs in HIV, BRF’s SPAR research led to the creation of innovative methods of testing for diseases of the male genital tract, including the prostate. Success and expertise in fertility research and treatment lead to BRF scientists implementing the first human egg donor program for stem cell research in September 2000.

WHY WE DO IT

BRF exists to pursue research with the most curative potential for diseases affecting millions of people today. A 200-fold growth since its inception testifies to the excitement and importance of program goals. Through continued education and intense research efforts, BRF will change the pace of progress for diseases that currently have no cure.

A moratorium on federal funding for crucial areas of biomedical research means public charities like BRF are the only means of bridging the gap between what the government supports and what people need. Now more than ever, BRF’s innovative, cost-effective research efforts are essential to the development of life-saving procedures and cures.

HOW WE DO IT

Bedford Research Foundation is a nonprofit, 501(c)(3) Massachusetts public charity. BRF has far lower operating costs than larger institutions, meaning more research results from every donation received. BRF relies on the insightful generosity of corporations, organizations and individuals to continue its vital work.

HOW YOU CAN HELP

Support BRF by joining a network of hundreds of donors helping to shape the future of science and medicine. Financial contributions can be designated for specific programs such as stem cell or prostate research, or can go toward general research and operating costs. Donations can be given in honor or in memory of a friend, colleague or family member. Volunteers, in-kind and other support are always welcome. If you would like to help, please visit www.bedfordresearch.org.

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.