Fight Aging! Newsletter, May 2nd 2016
This study is the first CAR T-cell trial to infuse patients with an even mixture of two types of T cells (helper and killer cells, which work together to kill cancer). With the assurance that each patient gets the same mixture of cells, the researchers were able to come to conclusions about the effects of administering different doses of cells.
In 27 of 29 participants whose responses were evaluated a few weeks after the infusion, a high-sensitivity test could detect no trace of their cancer in their bone marrow. The CAR T cells eliminated cancers anywhere in the body they appeared. Of the two participants who did n...
Source: Fight Aging! - May 1, 2016 Category: Research Authors: Reason Tags: Newsletters Source Type: blogs
The Scientist on BioViva's Initial Test of Human Gene Therapies
The Scientist has published a measured piece on the first results from BioViva's initial test of human gene therapy, telomerase and follistatin overexpression, and the broader context in which this single person test took place. The results indicate that the telomerase gene therapy most likely worked in the sense of delivering telomerase to a significant number of cells, including the immune cells used to measure average telomere length. That is an important thing to validate up front, before thinking about any sort of other outcomes, or expanding to a trial of some sort. Historically, gene therapies have proven to be high...
Source: Fight Aging! - April 25, 2016 Category: Research Authors: Reason Tags: Healthy Life Extension Community Source Type: blogs
Updates to DUX4 region on subtelomeric chromosome 4q
Sequence improvements and increased variant representation in the human genome reference assembly are priorities for the GRC. This blog post describes two updates in the recent GRCh38.p7 patch release affecting the DUX4 region located at the chromosome 4q sub-telomere: (1) a FIX patch correcting the chromosomal representation (KQ983257.1) and (2) a NOVEL patch representing a variant of the region (KQ983258.1). A third haplotype of DUX4 region which is represented in GRCh37 will be also described.The DUX4 region contains tandem arrays of a 3.3 Kb D4Z4 macrosatellite repeat located in the sub-telomer...
Source: GenomeRef - April 7, 2016 Category: Genetics & Stem Cells Source Type: blogs
The unknown impact of changing a person’s genetic makeup
In February 2015, the British Parliament approved the creation of a human embryo from the DNA of three people: mother, father and a donor mother. The modified in-vitro fertilization (IVF) technique, called mitochondrial replacement therapy (MRT), would help some mothers with known rare mitochondrial mutations avoid passing on unhealthy defects. These defects can cause severe or deadly diseases, which are often incurable, such as muscular dystrophy, heart and kidney disease, liver failure and severe muscle weakness. Approximately 99.9 percent of our DNA comes from our mother and father. The donor mother would provide the a...
Source: Kevin, M.D. - Medical Weblog - April 1, 2016 Category: Journals (General) Authors: Justin Morgan, MD Tags: Physician Genetics Source Type: blogs
Gene Editing Improves Mice With Duchenne Muscular Dystrophy
Duke researchers overcame a number of problems to deliver CRISPR-Cas9 genetic editing machinery into the muscle cells of living mice and treat a genetic disease. Another approach, which involves taking CRISPR directly to the affected tissues through gene therapy techniques, also faces challenges, particularly with delivery. In the new study, Duke University researchers overcame several of these obstacles by using a non-pathogenic carrier called adeno-associated virus, or AAV, to deliver the gene-editing system. Since Duchenne Muscular Dystrophy kills by a person's teens or twenties the high risk of in-place genetic editing...
Source: FuturePundit - January 1, 2016 Category: Research Authors: Randall Parker Source Type: blogs
Gene Therapy Treats Duchenne Muscular Dystrophy Dogs
Dogs lead the way. Due to its size, it is impossible to deliver the entire gene with a gene therapy vector, which is the vehicle that carries the therapeutic gene to the correct site in the body, Duan said. Through previous research, we were able to develop a miniature version of this gene called a microgene. This minimized dystrophin protected all muscles in the body of diseased mice. However, it took the team more than 10 years to develop a strategy that can safely send the micro-dystrophin to every muscle in a dog that is afflicted by the disease. The dog has a body size similar to that of an affected boy. Succe...
Source: FuturePundit - October 31, 2015 Category: Research Authors: Randall Parker Source Type: blogs
To the Edge of the Sky: the Fight to Save Children’s Lives and End Duchenne Muscular Dystrophy
To the Edge of the Sky documents four families fight to save their children's lives and end Duchenne Muscular Dystrophy.
Read the rest of the story... » (Source: Highlight HEALTH)
Source: Highlight HEALTH - May 8, 2015 Category: Consumer Health News Authors: Jenny Jessen Source Type: blogs
To the Edge of the Sky: the Fight to Save Children ’s Lives and End Duchenne Muscular Dystrophy
To the Edge of the Sky documents four families fight to save their children's lives and end Duchenne Muscular Dystrophy.
Read the rest of the story... » (Source: Highlight HEALTH)
Source: Highlight HEALTH - May 8, 2015 Category: Consumer Health News Authors: Jenny Jessen Source Type: blogs
The Year of Educational Choice: An Update
Back in February, I speculated that 2015 might be the “Year of Educational Choice” in the same way that the Wall Street Journal declared 2011 the “Year of School Choice” after 13 states enacted new or expanded school choice laws.
This year, in addition to a slew of more traditional school choice proposals, about a dozen legislatures considered new or expanded education savings accounts (ESAs). As I explained previously:
ESAs represent a move from school choice to educational choice because families can use ESA funds to pay for a lot more than just private school tuition. Parents can use the ESA funds for tutors, te...
Source: Cato-at-liberty - April 24, 2015 Category: American Health Authors: Jason Bedrick Source Type: blogs
Grafting Tissue Engineered Muscles into Mice
Tissue engineering of muscle continues to move forward, with a new approach here demonstrated in mice:
Tissue engineering of skeletal muscle is a significant challenge but has considerable potential for the treatment of the various types of irreversible damage to muscle that occur in diseases like Duchenne muscular dystrophy. So far, attempts to re-create a functional muscle either outside or directly inside the body have been unsuccessful. In vitro-generated artificial muscles normally do not survive the transfer in vivo because the host does not create the necessary nerves and blood vessels that would support the muscle...
Source: Fight Aging! - February 25, 2015 Category: Research Authors: Reason Tags: Daily News Source Type: blogs
FDA Approves 41 New Medicines in 2014, the Most Since 1996
The Food and Drug Administration (FDA) approved 41 new medicines in 2014, the most since 1996, when the agency approved a record 53. FDA approved 27 in 2013. The jump can be attributed to many new drugs for orphan diseases, which are rare conditions and disorders that affect fewer than 200,000 people in the U.S.
The spike in approvals is notable for a number of reasons.
For one, the pharmaceutical industry as a whole has been bouncing back from a number of patent losses on big name products over the last couple of years—the so-called patent cliff. While a number of large companies indeed will face stiff generic co...
Source: Policy and Medicine - January 5, 2015 Category: American Health Authors: Thomas Sullivan Source Type: blogs
Dame Kay Davies on her lifetime’s work in Duchenne muscular dystrophy
Recently, F1000 Publisher Kathleen Wets visited Dame Kay Davies in Oxford soon after she won the WISE Lifetime Achievement Award. The award celebrated her outstanding career in science, which inspired other women to follow in her footsteps. In Kathleen’s video, … Continue reading → (Source: Naturally Selected)
Source: Naturally Selected - December 10, 2014 Category: Biomedical Science Authors: Adie Chan Tags: Faculty Members Video Dame Kay Davies Duchenne Duchenne's muscular dystrophy WISE Lifetime achievement award Source Type: blogs
National Quality Forum Begins Annual Review of Quality Measures, Comments Open December 23 on the 202 Proposed Measures
On Monday, the Measure Applications Partnership (MAP) began its annual review of performance measures that the Centers for Medicare and Medicaid Services (CMS) is considering for use in 20 federal health programs. The 202 measures considered by the group have been made public (view the PDF), and will be available for review and comment beginning December 23, 2014.
Established by the National Quality Forum (NQF) in 2011, MAP is a forum of approximately 150 healthcare leaders and experts, representing nearly 90 private-sector organizations. MAP comprises consumers, purchasers, labor, health plans, cl...
Source: Policy and Medicine - December 5, 2014 Category: American Health Authors: Thomas Sullivan Source Type: blogs
Meet Jennifer Doudna
Credit: Jennifer Doudna
Jennifer Doudna
Fields: Biochemistry and structural biology
Studies: New genome editing tool called CRISPR
Works at: University of California, Berkeley
Raised in: Hilo, Hawaii
Studied at: Pomona College, Harvard University
Recent honors: Winner of the Lurie Prize in the Biomedical Sciences , an annual award that recognizes outstanding achievement by promising scientists age 52 or younger
If she couldn’t be a scientist, she’d like to be: A papaya farmer or an architect
Jennifer Doudna likes to get her hands dirty. Literally. When she’s not in her laboratory, she can often be found amid glossy ...
Source: Biomedical Beat Blog - National Institute of General Medical Sciences - November 6, 2014 Category: Research Authors: Srivalli Subbaramaiah Tags: Chemistry and Biochemistry Profiles Structural Biology Source Type: blogs
Another Method of Restoring Activity in Old Muscle Stem Cells
Stem cell activity progressively declines with age, leading to lapsed tissue maintenance and increasing dysfunction and frailty. The role of stem cells is after all to replenish tissues, to provide a supply of fresh cells to replace those lost by the normal turnover, as well as issuing signals that spur other cell types into repair and regenerative efforts. Why does stem cell activity diminish? It is most likely an evolved reaction to rising levels of cellular damage, and serves to reduce the risk of cancer: our long life span in comparison to other primates is a balancing act between death by cancer on the one hand and de...
Source: Fight Aging! - September 8, 2014 Category: Research Authors: Reason Tags: Medicine, Biotech, Research Source Type: blogs
