What is the germline gene therapy?
Germline gene therapy is when DNA is transferred into the cells that produce reproductive cells, eggs or sperm, in the body. This type of therapy allows for the correction of disease-causing gene variants that are certain to be passed down from generation to generation.
When was germline gene therapy invented?
The concepts of gene therapy arose initially during the 1960s and early 1970s whilst the development of genetically marked cells lines and the clarification of mechanisms of cell transformation by the papaovaviruses polyoma and SV40 was in progress.
What is in vivo and ex vivo gene therapy?
Ex vivo gene therapy involves removing cells from your body, modifying them, and placing them back into your body. With in vivo gene therapy, new genes are inserted directly into your body. Both methods have risks and side effects associated with them.
Which disorder has been treated by in vivo gene therapy?
In this review, we cover both preclinical animal studies and clinical human trials that have used ex vivo gene therapy to treat neurological disorders with a focus on Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, ALS, and stroke.
Which is an example of germline gene therapy?
This type of gene modification is still in the early stages of human medical intervention but examples do exist, such as in cows modified to have elevated levels of milk production or the capability to secrete human hormones, or “knockin” and “knockout” mouse models, used for decades to elucidate gene function.
Why is germline gene therapy controversial?
This approach is known as germline gene therapy. The idea of these germline alterations is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known.
Why is germline gene therapy illegal?
Because people who would be affected by germline gene therapy are not yet born, they can’t choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.
Who discovered germline therapy?
French Anderson, MD, was “dubbed ‘the father of gene therapy’ after a team he led in 1990 cured a hereditary disease of the immune system in a 4-year-old girl.” That’s not quite the way it happened.
What are the 2 types of gene therapy?
There are two different types of gene therapy depending on which types of cells are treated:
- Somatic gene therapy: transfer of a section of DNA to any cell of the body that doesn’t produce sperm or eggs.
- Germline gene therapy: transfer of a section of DNA to cells that produce eggs or sperm.
What is the difference between in vitro and ex vivo?
In Vitro vs.
The cells and tissues for ex vivo experiments are taken from a living organism, whether donated or harvested (e.g., hair follicles, skin explants). Meanwhile, for in vitro tests, cells are obtained from repositories and cultured to create the necessary model (e.g., reconstructed human epidermis).
What is a disadvantage of in vivo gene therapy?
Disadvantages of in vivo gene transfer are the possibility that the introduction and expression of virus proteins may activate endogenous pathogenic viruses (Romano et al., 2000). It has also been suggested that replication-competent recombinant viruses may form due to random mutagenesis.
Is germline gene therapy allowed?
Federal law prohibits the use of federal funds for research on human germline gene therapy. Germline gene editing is banned in the United States by acts of Congress although there is no federal legislation that dictates protocols or restrictions regarding human genetic engineering.
Is germline gene therapy safe?
The earliest studies showed that gene therapy could have very serious health risks, such as toxicity, inflammation, and cancer. Since then, researchers have studied the mechanisms and developed improved techniques that are less likely to cause dangerous immune reactions or cancer.
Where is germline gene therapy legal?
In China and the United Kingdom, human germline genome editing is permitted. In China, heritable human genome editing is prohibited by the Ethical Guiding Principles on Human Embryonic Stem Cell Research (2003).
Is germline gene therapy legal?
Which type of gene therapy is the most controversial?
Germline gene therapy has been more controversial than somatic gene therapy because it involves deliberate, inheritable changes in the genome. Germline gene therapy poses risks not only to patients but also to future generations.
Why in vivo is better than in vitro?
The use of animals in in vivo studies addresses many of the shortcomings of in vitro studies. Scientists can better evaluate the safety, toxicity and efficacy of a drug candidate in a complex model. Moreover, advances in gene editing have helped scientists replicate human diseases in animals with high accuracy.
What is an example of ex vivo?
Testing the effect of compounds on skin biopsies is an example of ex vivo research, while isolating the primary cells from that biopsy and creating a 3D cell culture model is an example of in vitro research. Both use human tissues, but the former is a more complex and translational environment for drug testing.
Why are people against gene therapy?
Three arguments sometimes raised against gene therapy are that it is technically too dangerous, that it discriminates or invites discrimination against persons with disabilities, and that it may be becoming increasingly irrelevant in some cases.
Is germline gene therapy unethical?
Ethical Concerns for Germline Gene Editing
The potential benefits and potential harms for both the edited individual and the larger population may not be fully known or appreciated within the lifetime of the treated patient, and many generations would need to be studied to understand the long-term effects.
Which countries have banned germline editing?
Four countries (Albania, Bahrain, Belarus, and Croatia) have policy documents that appear to prohibit all research involving human embryos. All four are categorized as prohibiting germline genome editing on the basis of their prohibition of all human embryo research.
Why is it called a germline?
Germ line. Some organisms reproduce sexually and need special sex cells named germ cells to pass their genomes from parents to offspring. Germ cells are known as egg cells in females and sperm cells in males. And together, they make up the germ lines of the organism.
How many countries have banned germline editing?
Twenty-three countries explicitly prohibit germline genome editing research; 11 permit it; and six are indeterminate.
What are the disadvantages of in vivo testing?
Some of the disadvantages of in vivo studies include: Whole, living organisms are used, which can prove to be unethical if harm or distress is caused. It is a lot harder to control every variable, so the results may not be reliable nor applicable to a wider population. Can sometimes be expensive.
What is the difference between vivo and vitro?
An in vitro study occurs in a controlled environment, such as a test tube or petri dish. In vivo is Latin for “within the living.” It refers to tests, experiments, and procedures that researchers perform in or on a whole living organism, such as a person, laboratory animal, or plant.