Myostatin Inhibiting Gene Therapy Success in Monkeys

One person in a million has natural myostatin inhibition genes

Work was done on Macaque monkeys. Commonly scientific work is on rhesus macaques.

Researchers at Nationwide Children’s Hospital have shown that a gene delivery strategy that produces follistatin — a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in skeletal muscle — directly to the quadriceps of non-human primates results in long-term gene expression with muscle enhancing effects, including larger muscles with greater strength. The muscles were 15% bigger, 78% stronger and the effect lasted for the 15 month study with no negative health effects. The treatment produced no obvious negative side-effects and human clinical trials are expected to start next year.

Myostatin inhibition occurs naturally in about one in one million people. Studies with mice indicate myostatin inhibition is four times more effective than high doses of steroids. Myostatin inhibition can have various health benefits and not just performance enhancment. It can be used to counter the muscle wasting in old people and help old people stay mobile longer. Mobile people stay healthier longer. Another potential benefit is help people with weight control and reduction of obesity. Muscles consume more calories than fat so someone with Myostatin inhibition should find it easier to manage their weight. Having fewer obese people should improve public health and could also help with national medical costs.

A previous study of myostatin inhibitors on mice has shown protection against obesity and hardening of the arteries

Obesity increases the risk of atherosclerosis, which accounts for 75% of all cardiovascular events, such as heart attacks and strokes. The mice with deleted myostatin gene had much less body fat and 30 percent lower fasting blood sugar and 80% lower fasting insulin levels, showing a reduction in obesity and a strong resistance to developing diabetes, the authors reported. They also had 50 percent lower low-density-lipoprotein (“bad”) cholesterol and 30 to 60 percent lower levels of total cholesterol and triglycerides (fats in the blood), respectively. These results indicate protection against the development of atherosclerosis.

So safe (potentially health improving) and very effective muscle enhancement appears to be close. At the end of this article is other option which is already being used by people now (SARMs – produce a steroid like effect that only goes to the muscles).

Estimates are that 7 million people take steroids to boost their muscles (in spite of the health risks) and most do it for enhanced appearance and not for sports.

Steroid like effects with no health risks and potentially health benefits would seem likely to be very widely used. If the protection against obesity and artery hardening holds up in human trials then it would make sense for everyone to have the myostatin inhibition treatment to protect and cardiovascular disease and death which is the number one cause of death. Abut 30% of all deaths are cause by heart disease

This more recent study produced similar results in non-human primates, in a translational study to demonstrate efficacy in safety in a species more closely related to humans. Non-human primates that received the injection of the follistatin transgene experienced pronounced and durable increases in muscle size and strength. Muscle growth occurred for 12 weeks after treatment, after which time the growth rates appeared to stabilize and were well tolerated, with no adverse events noted over the course of the 15-month study.

“Our studies indicate that this relatively non-invasive approach could have long-term effects, involve few risks and could potentially be effective in various types of degenerative muscle disorders including multiple forms of muscular dystrophy,” said the study’s corresponding author, Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy of The Research Institute at Nationwide Children’s Hospital.

Times UK has more info

After the treatment, the monkeys’ leg muscles grew steadily, and were 15 per cent bigger in circumference on average after eight weeks.

Using electrical muscle stimulation, the scientists showed that the treated legs were also significantly stronger than the untreated legs. In one monkey, the treated leg was 78 per cent stronger. .

The enhanced size and power were retained 15 months after the treatment and caused no apparent health problems.

Follistatin Gene Delivery Enhances Muscle Growth and Strength in Nonhuman Primates

Antagonists of myostatin, a blood-borne negative regulator of muscle growth produced in muscle cells, have shown considerable promise for enhancing muscle mass and strength in rodent studies and could serve as potential therapeutic agents for human muscle diseases. One of the most potent of these agents, follistatin, is both safe and effective in mice, but similar tests have not been performed in nonhuman primates. To assess this important criterion for clinical translation, we tested an alternatively spliced form of human follistatin that affects skeletal muscle but that has only minimal effects on nonmuscle cells. When injected into the quadriceps of cynomolgus macaque monkeys, a follistatin isoform expressed from an adeno-associated virus serotype 1 vector, AAV1-FS344, induced pronounced and durable increases in muscle size and strength. Long-term expression of the transgene did not produce any abnormal changes in the morphology or function of key organs, indicating the safety of gene delivery by intramuscular injection of an AAV1 vector. Our results, together with the findings in mice, suggest that therapy with AAV1-FS344 may improve muscle mass and function in patients with certain degenerative muscle disorders.

Supplemental Material

Supplemental material in a 6 page pdf

The treatment did not cause sperm or menstrual cycles to go outside of normal ranges.

Fig. S1. MCK-FS causes myofiber hypertrophy and predominantly affects fast-twitch type 2 myofibers in the quadriceps muscle.
Fig. S2. Morphological effects on sperm from male cynomologus macaques treated with AAV1-FS344.
Table S1. Menstrual cycles in untreated control and AAV1-FS344–treated cynomolgus macaques.
Table S2. Sperm motility and morphology of untreated control and AAV1- FS344–treated cynomolgus macaques.
Table S3. Primer and probe sets used in quantitative polymerase chain reaction (QPCR) for vector genome quantification.

Seperate Product/Method for Potentially Safe Muscle Boosting
Steroid like effects that are theorized to be safer are available online from SARMs.

SARMs work similarly to testosterone but in a more targeted way. “They are effective by binding to the steroid receptor in only specific tissue, like muscle,” says Evans, who is also a scientific advisor to GTx, a company developing the drugs. “They are not steroid drugs, but they produce the anabolic effect of the steroids.” GTx, based in Memphis, TN, has shown in a clinical trial that one compound being developed for muscle wasting and bone loss can significantly boost lean muscle mass in older people.


The current black market use of SARMs (before clinical trials are done, phase 3 clinical trials not started) via online purchase and the online sale of fake myostatin inhibitors and fake SARMs shows the massive demand that will be there for the real thing. Especially after they do clear clinical trials for Muscle wasting diseases. I expect to be having some type of myostatin inhibitor treatment within ten years. Maintain muscles before getting too hold and maintaining as I get older, less risk of falling and breaking a hip later, heart health, weight control. do not care about the sports aspect. The real sports competitors will doing this and ten other riskier things. I had LASIK done over ten years ago and have not regretted it. Was able to get 20-15 vision (so better than the old good vision standard of 20-20).

The “clean” sports leagues will look like childrens little league compared to the open enhancement leagues.

2020 expectation:
SARMs and Myostatin inhibition widespread (tens of millions)
20-5 vision common from advanced LASIK and other treatments
Next generation cognitive enhancement drugs
robotic and cyber enhancements
Augmented reality vision (goggles/visors)
Mind machine interfaces

The next level after those will be transgenic (gorilla strength, 40 mph speed, super leaping, cognitive remodelling – stem cells, gene therapy, superdrugs 300IQ-1000IQ and may more), molecular nanotechnology enhancement. Although the performance enhancement is interesting, it will be the enhancements for
– super immune systems
– life extension and health enhancement
– radiation and other resistence
– cognitive enhancement
– regeneration

that will be the big impacters of civilization.

human enhancement overview

whole body gene therapy progress

Japanese create transgenic monkeys

Transgenic genetic engineering update