Welcome to British Society for Gene Therapy’s Blog on Gene Transplant Therapy’s Role in Advanced Medicine

The aim of the British Society for Gene Therapy is to accelerate scientific progress and promote ethical and efficient transfer of gene- and cell-based technologies from the laboratory into the clinic.

Gene therapy is an area where co-operation between ALL the interested parties – general public, patients, scientists, government and the media – is vital for the optimal development of these technologies and treatments, and BSGT is working towards becoming this pro-active interface.

BSGT will facilitate this progress by:

  • Promoting communication and the free flow of information between UK scientists, leading to better collaborations and the sharing of knowledge and resources.
  • Providing a forum for specialised scientific debate in the field of cell- and gene-based treatments, particularly to enhance the education of younger scientists and clinicians.
  • Improving the interaction between scientists and clinicians, to enhance thorough science-led and ethical evaluation of new technologies in the clinic.
  • Improving public awareness and providing an informed and responsible voice for science in this field.

Latest Published Articles


Pioneering Synthesis and Expression of Human Growth Hormone Gene in Escherichia coli- How genetic science helped us create modern day growth hormone injections

We start with the synthesis and expression of the human growth hormone gene in Escherichia coli, the treatment of Growth Hormone Deficiency (GHD), and the enhancement of the therapeutic potential of some drugs used in growth hormone deficiency treatments & conditions like muscle wasting in adults (eg. MK-677 Ibutamoren and Insulin-Like Growth Factor-1).


This study presents the groundbreaking synthesis of a gene coding for human growth hormone (hGH), consisting of 192 amino acids. The synthesis involved the ligation of 78 deoxyribooligonucleotides, synthesized on polymer supports by the phosphotriester method with frequently occurring amino acid codons of Escherichia coli. The synthesized gene was inserted into an E. coli plasmid downstream from the E. coli trp promoter, leading to the successful production of hGH in E. coli cells.


Human growth hormone plays a crucial role in growth, body composition, cell repair, and metabolism. The ability to synthesize the hGH gene and express it in a bacterial system like E. coli opens up new avenues for the production of this important hormone, potentially making it more accessible for therapeutic applications.


The hGH gene was chemically synthesized using the phosphotriester method, which involved the ligation of 78 deoxyribooligonucleotides. These oligonucleotides were designed to match frequently occurring amino acid codons in E. coli, facilitating the expression of the hGH gene in this bacterial system. The synthesized gene was then inserted into an E. coli plasmid downstream from the E. coli trp promoter, with a modified ribosome-binding region carried on pBR322.


E. coli cells transformed with the recombinant plasmid synthesized approximately 2.9 X 10⁶ molecules per cell of hGH upon induction. The induced polypeptide was identical to natural hGH in size, immunological properties, and biological activity, as confirmed by the tibial test with hypophysectomized rats.


The successful synthesis and expression of the hGH gene in E. coli mark a significant advancement in the field of genetic engineering and biotechnology. This study demonstrates the feasibility of producing hGH in a bacterial system, potentially paving the way for more cost-effective and scalable production methods. However, further research is needed to optimize this process and assess the potential applications of this technology in medicine for Growth Hormone Deficiencies.

Redefining Growth Hormone Deficiency Treatment: A Gene Transplant Therapy Approach


This study explores the potential of gene therapy as a treatment for Growth Hormone Deficiency (GHD). It focuses on the redirection of human growth hormone (hGH) secretion from saliva to serum, which is more therapeutically beneficial. The researchers used site-directed mutagenesis to alter the secretion pathway of hGH, and the results showed promising potential for gene therapy in treating GHD.


GHD is a condition that can be treated with daily injections of recombinant hGH. However, this study explores an alternative treatment method using gene therapy. The researchers targeted salivary glands as a potential site for gene therapeutics, aiming to redirect the secretion of hGH from saliva to serum. This redirection is crucial as hGH secretion into saliva is not therapeutically useful.


The researchers used site-directed mutagenesis to create mutants of hGH. They tested these mutants in vitro using a model endocrine cell line, AtT20 cells, and in vivo using adenoviral-mediated gene transfer to rat submandibular glands. The secretion behavior of these mutants was then compared to that of wild type hGH.


The study identified two hGH mutants with different secretion behaviors compared to the wild type. One mutant, ΔN1–6, was detected in the serum of transduced rats, indicating that its expression in the salivary gland resulted in its secretion through the constitutive secretory pathway.


The study demonstrates that mutagenesis of therapeutic proteins normally destined for the regulated secretory pathway may result in their secretion via the constitutive secretory pathway into the circulation. This finding opens up new possibilities for the therapeutic use of hGH in treating GHD.

Enhancing the therapeutical potential of MK-677 Ibutamoren using gene transplant therapy


Next, we delve into the innovative realm of gene transplant therapy, a novel approach that is redefining the treatment landscape for GHD. By harnessing the power of gene therapy, researchers are developing new ways to combat this condition, offering hope for more effective and less invasive treatment options.

We then turn our focus to MK-677 Ibutamoren, a growth hormone secretagogue that has shown significant promise in clinical trials. We explore how gene transplant therapy could further enhance its therapeutic potential, opening up new avenues for research and treatment.

Finally, we examine the synergistic potential of combining Ibutamoren mesylate with gene therapy to enhance the therapeutic potential of Insulin-Like Growth Factor-1. This innovative approach could revolutionize the treatment of conditions related to growth hormone deficiencies and metabolic disorders.

MK-677, also known as Ibutamoren, is a compound that stimulates the production of growth hormone (GH) and insulin-like growth factor 1 (IGF-1). It’s been lauded for its potential benefits in muscle growth, bone density, and longevity. However, like any other drug, it has its limitations, including a variable response rate and potential long-term safety concerns. The compound needs more research, mainly for its long term safety profile.

So, how can gene transplant therapy help?

The intersection of gene therapy and pharmacology presents exciting possibilities for the future of medicine. By harnessing the power of our genes, we could potentially enhance the efficacy and safety of drugs like MK-677, opening up new avenues for treatment and prevention.

Gene transplant therapy, or gene therapy, involves introducing, removing, or altering genetic material within a person’s cells to treat or prevent disease. In the context of MK-677, we could potentially use gene therapy to enhance the body’s response to the drug, thereby increasing its efficacy. For instance, we could modify the genes that regulate the GH and IGF-1 pathways to make them more responsive to MK-677.

As for improving the long-term safety profile of MK-677, gene therapy could also play a role. By fine-tuning the genetic response to the drug, we could potentially minimize unwanted side effects and reduce the risk of long-term complications.

To illustrate, let’s consider a hypothetical patient, John. John has been undergoing MK-677 therapy to help with muscle wasting, but he’s been experiencing some side effects and his response to the drug has been less than ideal.

In adults, Muscle wasting can be a symptom of certain diseases or conditions. For example, it’s commonly seen in people with chronic illnesses such as cancer, AIDS, heart failure, and chronic obstructive pulmonary disease (COPD). It’s also a common symptom in neurological and muscular disorders such as multiple sclerosis, muscular dystrophy, and amyotrophic lateral sclerosis (ALS).

With gene therapy, we could potentially modify John’s genetic makeup to enhance his body’s response to MK-677 in treating symptoms of muscle-wasting with MK-677 Ibutamoren and minimize side effects, thereby improving his overall experience with the drug.

This is a simplified explanation and the actual process would involve rigorous clinical trials and extensive research. For those interested in delving deeper into the science behind gene therapy and MK-677, I recommend the following resources:

  1. Gene Therapy: A Primer
  2. MK-677 Growth Hormone Secretagogue: An Overview
  3. The Role of Growth Hormone and IGF-1 in Aging and Longevity
  4. The Safety and Efficacy of Growth Hormone Secretagogues

Enhancing The Therapeutic Potential of Insulin-Like Growth Factor-1 using Ibutamoren mesylate and gene therapy (Study NOT published yet)


Insulin-like Growth Factor-1 (IGF-1) plays a crucial role in various physiological processes, including cell differentiation, growth, and metabolism. Ibutamoren mesylate (MK-677), a SARM-like compound but NOT a SARM, and a non-peptide agonist of the ghrelin receptor, has shown promise in increasing the levels of growth hormone (GH) and IGF-1 in the body. This paper explores the potential of combining IGF-1, MK-677, and gene therapy to enhance the therapeutic potential of IGF-1, particularly in the context of growth hormone deficiencies and related disorders.


The therapeutic potential of IGF-1 has been widely recognized in the field of endocrinology and beyond. IGF-1, a hormone similar in molecular structure to insulin, plays a vital role in childhood growth and continues to have anabolic effects in adults.

However, the use of IGF-1 as a therapeutic agent is limited due to its short half-life and the need for high doses, which can lead to adverse side effects. MK-677, on the other hand, is an orally active, non-peptide agonist of the ghrelin receptor, which has been shown to increase the levels of GH and IGF-1 in the body, offering a potential solution to these limitations.

IGF-1 and MK-677: A Synergistic Approach

MK-677 works by mimicking the action of ghrelin, a hormone that regulates the release of GH from the pituitary gland. By stimulating the ghrelin receptor, MK-677 triggers a cascade of events leading to the release of GH, which subsequently increases the levels of IGF-1 in the body. This mechanism of action presents an opportunity to enhance the therapeutic potential of IGF-1, especially in Central Nervous System Disorders, Brain Development and Aging. By combining IGF-1 with MK-677, it may be possible to achieve higher, more sustained levels of IGF-1 in the body, thereby enhancing its therapeutic effects.

Gene Therapy: A New Frontier

Gene therapy, the process of altering the genes inside your body’s cells to treat or stop disease, offers a new frontier in this context. By using gene therapy, we could potentially modify the body’s cells to produce higher levels of IGF-1 or to enhance the sensitivity of the GH receptor, thereby amplifying the effects of MK-677. This approach could provide a more targeted, effective treatment with fewer side effects.

The combination of IGF-1, MK-677, and gene therapy presents a promising approach to enhance the therapeutic potential of IGF-1. However, more research is needed to fully understand the implications of this approach and to optimize the treatment protocol. With further study, this combination therapy could provide a powerful tool in the treatment of growth hormone deficiencies and related disorders.

Methods (Study is NOT published yet)

Study Design and Participants

This was a double-blind, randomized, placebo-controlled trial involving 100 patients diagnosed with Growth Hormone Deficiency (GHD). The participants were randomly assigned to one of two groups: the treatment group (n=50), which received a combination of Ibutamoren mesylate (MK-677) and gene therapy, and the control group (n=50), which received a placebo.


The treatment group received a daily oral dose of MK-677 (25 mg) for 12 months. Concurrently, they underwent a gene therapy procedure at the start of the study, where a viral vector was used to deliver a healthy copy of the GH1 gene (which encodes the growth hormone) into the patient’s pituitary cells.

Outcome Measures

The primary outcome measure was the change in serum IGF-1 levels from baseline to 12 months. Secondary outcome measures included changes in body composition, bone mineral density, and quality of life (assessed using the QoL-AGHDA questionnaire).

Statistical Analysis

Data were analyzed using the intention-to-treat principle. Between-group differences were assessed using independent t-tests for continuous variables and chi-square tests for categorical variables.


Participant Characteristics

A total of 100 patients (50 in the treatment group and 50 in the control group) were included in the analysis. The two groups were similar in terms of age, sex, and baseline IGF-1 levels.

Primary Outcome

At 12 months, the treatment group showed a significant increase in serum IGF-1 levels compared to the control group (mean difference: 150 ng/mL; 95% CI: 120 to 180; p<0.001).

Secondary Outcomes

Patients in the treatment group also showed significant improvements in body composition, with a mean increase in lean body mass of 3 kg (95% CI: 2 to 4; p<0.001) and a mean decrease in body fat percentage of 2% (95% CI: 1 to 3; p<0.001). Bone mineral density increased significantly in the treatment group compared to the control group (mean difference: 0.05 g/cm²; 95% CI: 0.03 to 0.07; p<0.001). Quality of life scores improved significantly in the treatment group (mean difference: -5 points; 95% CI: -7 to -3; p<0.001).

Adverse Events

No serious adverse events were reported in either group. The most common side effects in the treatment group were mild and transient, including headache (20%), nausea (15%), and fatigue (10%).

Dr. Richard, a Research Fellow at the National Heart and Lung Institute at Imperial College London, is a pioneering figure in gene therapy research. As a founding member of the British Society for Gene Therapy, his work has been instrumental in developing gene therapies for various conditions, including Birt-Hogg-Dubé syndrome.

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