What are the current treatments for genetic diseases?

There are generally three types of common genetic disorders: monogenic disorders, polygenic disorders, and chromosomal disorders. What are the current methods of genetic diseases?

Because the essence of genetic diseases is genetic abnormality, ordinary medical methods are helpless for the vast majority of genetic diseases, so by replacing or correcting defective genes, or by regulating gene expression, the genetic disease is carried out, which is the gene. In 1990, American scientists took the lead in adopting this method and successfully completed the world's first genetic clinical trial for genetic diseases.

At present, one of the methods of genetic diseases is gene modification and gene replacement, and the other is gene enhancement and gene inactivation.

What is an underlying medical condition? Underlying diseases are relative to the current major diseases, such as cerebrovascular diseases, including cerebral infarction, cerebral hemorrhage, cerebral infarction is currently the main disease, and its corresponding underlying diseases mainly include hypertension, diabetes, hyperlipidemia, hyperhomocysteinemia, heart disease, and others, such as long-term chronic inflammation, these underlying diseases will increase the risk of stroke, they should be called underlying diseases. Coronary heart disease is now the main disease, and its underlying diseases are hypertension, hyperlipidemia, diabetes, etc., which are called underlying diseases.

The :* and prevention of polygenic genetic diseases, to inherit diseases, the defects that have occurred should be corrected from the genetic level or the chromosomal level, which is called genetics, which belongs to the category of genetic engineering. But the gene ** in theory、

There are still great technical difficulties, and there is no clinical application at present. At present, the only way to do genetic diseases is to control environmental conditions (such as dietary composition, etc.) to regulate metabolic processes and prevent the appearance of symptoms under the premise of early diagnosis, which is called "environmental engineering." At present, the ** that can be applied to environmental engineering ** includes dietary control ** (such as phenylketonuria fed with low or no phenylketon-free yogurt powder), drugs ** (such as vitamin B6** B6 dependence, allopurinol ** gout, etc.), surgery ** (such as splenectomy ** hereditary spherocytosis).

Enzyme supplementation (eg, allogeneic bone marrow transplantation** Gaucher disease) and symptomatic (eg, seizures with antiepileptic drugs to control phenylketonuria). Although environmental engineering can alleviate or eliminate the symptoms of some genetic diseases, which is beneficial to individuals, the result is that patients with disease-causing genes not only survive, but even continue to reproduce, and these patients could have been eliminated naturally without **, at least not reproduced. Therefore, the impact of environmental engineering on the whole human race may be detrimental, and it will increase the frequency of disease-causing genes in the population from generation to generation, leading to an increase in the incidence of genetic diseases.

At present, there is no effective means for genetic diseases, because genetic diseases are special diseases caused by genetic defects, and this disease will have different methods due to the patient's time of illness, the location of the disease, the degree of damage and the tolerance to adverse reactions. But even if it is **, it can only play a relieving role, it is impossible to ****. Therefore, patients with genetic diseases are advised to actively cooperate with doctors**, pay attention to diet, and pay attention to rest.

Although it cannot be the root cause of the disease, it at least has a relieving effect and reduces the suffering caused by the disease.

Gene refers to the new biomedical technology that introduces human normal genes or genes with the best effect into human target cells in a certain way to correct gene defects or play a leading role, so as to achieve the purpose of disease. A gene is the basic functional unit that carries the genetic information of an organism and is a specific sequence located on a chromosome. The introduction of exogenous genes into biological cells must rely on certain technical methods or vectors, and the current methods of gene transfer are divided into biological methods, physical methods and chemical methods.

Adenovirus vectors are one of the most commonly used viral vectors for genetically **.

The target cells of genes are mainly divided into two categories: somatic cells and germ cells, and the current gene** is limited to somatic cells. Genes are currently predominantly those diseases that pose a serious threat to human health, including:

Genetic diseases (such as hemophilia, cystic fibrosis, familial hypercholesterolemia, etc.), malignant tumors, cardiovascular diseases, infectious diseases.

Gene ** refers to the use of DNA recombination amplification to repair or reconstruct diseased genes or defective genes in the patient's cells, so as to achieve the purpose of restoring the function of cells to normal, which is gene**. The maternal gene is generally a genetic disease by introducing a reconstituted circular DNA molecule into the body so that its expression products can function.

Gene therapy refers to the introduction of exogenous normal genes into target cells to correct or compensate for diseases caused by defective and abnormal genes in order to achieve the purpose. It also includes the application of transgenic and other technologies, that is, the introduction of foreign genes into the appropriate recipient cells of patients through gene transfer technology, so that the products manufactured by foreign genes can be used for a certain disease. Broadly speaking, genes can also include measures and new technologies for certain diseases taken from the DNA level.

Genetic disorders are the correction of defects that have occurred at the genetic level or at the chromosomal level. However, there are still great difficulties in the theory and technology of gene **, and there is no clinical application at present. At present, the only way to do genetic diseases is to control environmental conditions such as dietary composition and other conditions to regulate metabolic processes and prevent the appearance of symptoms under the premise of early diagnosis.

Genes** are divided into two types by genetic manipulation. One is genetic modification and gene replacement, which corrects the abnormal sequence of the defective gene and precisely repairs the defective gene in situ without any changes to the genome. Allogeneic genes are recombined at specific sites through homologous recombination, that is, gene targeting technology, so that the defective genes can be specifically repaired in situ.

the other is gene enhancement and gene inactivation, which does not remove abnormal genes, but introduces foreign gene expression normal products, thereby compensating for functions such as defective genes; or specifically block the translation or transcription of certain genes to inhibit the expression of certain abnormal genes.

Commonly used gene transfer methods include: recombinant virion-guided gene transfer, chemical reagent-guided gene transfer, shock-guided gene transfer, direct DNA injection and DNA particle injection. The methods of gene ** can be divided into two types: in vitro** and non-in vitro**.

Genetics is an emerging approach that aims to develop disease by modifying or repairing a patient's genes. The core idea of genetics is to use genetic engineering technology to introduce specific genes into the patient's cells to repair or change the function of abnormal genes.

Genes can be divided into three main types:

Gene replacement: This method aims to replace defective or abnormal genes by introducing a normal gene into a patient's cells. This can be done by introducing normal genes into the patient's cells through a vector (e.g., premature hail virus) that enables them to produce normal proteins or functions.

Gene repair: This method aims to repair abnormal genes present in the patient's body. By using genetic technologies such as CRISPR-Cas9, it is possible to perform precise and repair of the patient's genes directly to restore their normal function.

Gene targeting: This method works by interfering with or inhibiting the expression of specific genes for disease. This can be achieved through the use of RNA interference technology or gene-targeted drugs to block or inhibit the expression of abnormal genes.

Genomics is an important part of personalized medicine that can provide new options for patients with genetic diseases, cancer, immune system disorders, and more. However, Geno** is still in the research and development stage, and further research and clinical trials are needed to verify its safety and efficacy.

Advantages: Gene is a potential means, with the development of science and technology, gene will make greater progress and will be widely used. Diseases that have been genetically engineered** include hemophilia, severe anemia, arthritis, cardiovascular disease, cancer, and even intractable diseases such as AIDS.

Drugs that are lacking, such as artificial insulin, can be produced.

Disadvantages: The target gene may produce harmful gene mutations, the target gene generally uses liposomes as the carrier, the gene introduction efficiency is low, and there is no targeting, the adenovirus is used as the vector, the introduction efficiency is high, but it will cause a serious immune response, and may also cause cells other than the target cell to be infected with the virus, and may even infect other individuals or spread the virus to the environment, endangering the health of human Zhikai.

Genetics is an emerging method that can be used for some genetic diseases and diseases such as cancer. However, it also has some drawbacks and challenges:

Safety Concerns: Genes involve modification of a patient's genes or the introduction of foreign genes, which may lead to an unacceptable risk of premature hail. For example, it may cause an immune, inflammatory, or cytotoxic reaction, among other things.

Uncertainty of effect: The efficacy of gene** may vary due to individual differences, and some patients may not be sensitive or ineffective to **. In addition, the effects of genes may be temporary and need to be maintained regularly.

Lack of an effective delivery system: Delivering genetically ** drugs into specific tissues or cells is a challenge. At present, the lack of an effective delivery system limits the application of genes.

High cost: Genetics is a complex and expensive method, including the cost of genetic modification, drug preparation, delivery systems, etc. This makes genetics** less accessible to some patients.

It should be pointed out that with the continuous progress of science and technology, the above problems may be solved or improved. However, at present, Genomic** is still in the research and development stage, and more clinical trials and validation are needed to ensure its safety and efficacy.

Genomics is an emerging approach that is still in the research and development stage. While genetic** has great potential, there are also some potential risks and challenges.

Some of the possible risks include:

Immune response: The patient's immune system may react to the vectors used in **, such as viruses, leading to inflammation or other adverse reactions.

Poor effect: The effect of genes may be limited by many factors, such as the stability of gene expression, the conversion rate of cells, etc.

Genetic abrupt changes: Genes** may cause unintended genetic mutations that may cause patients to develop other diseases or other adverse reactions.

Moral and social issues: Genetics involves a number of ethical and social issues, such as privacy, fairness, and impartiality.

Overall, genetics is a promising approach, but more research and clinical trials are needed before implementation to ensure its safety and efficacy. At the same time, strict regulatory mechanisms and ethical frameworks need to be established to ensure that the implementation of genetics is safe, secure, and fair.

A method of introducing normal genes into hematopoietic stem cells or other tissue cells to correct their specific genetic defects, so as to achieve the best purpose.

Genetics is a new technology and the only effective method for many diseases. For those genetic diseases, inserting functional gene fragments can help some diseases, it can not only improve the lifelong protein replacement method, but even directly ** these diseases. For cancer genes, gene fragments can be introduced into cancer cells to kill cancer cells with minimal collateral damage to normal tissues.

In the brain, gene expression products can directly cross the blood-brain barrier. However, exogenous protein therapy is difficult to achieve, especially for diseases where the target is intracellular. Genetic IP overcomes these shortcomings and becomes an effective intracellular method.

In addition, genes can be stably expressed in vivo without the need for frequent monitoring. The length of time it is expressed depends on the nature of the transgenic fragment and the type of vector.

In order to avoid the risk of gene **, before being applied to the clinic, it is necessary to ensure the absolute safety of the transfer-expression system, so that the new gene will not harm the cell and the human body itself after the host cell is expressed, and will not cause the activation of oncogenes and the inactivation of anti-tumor genes, etc., especially when the reverse transcription vector is used for gene transfer, similar studies must be carried out in human bone marrow cells, mice and primates before being applied to the human body to ensure the safety of the first generation.