BioTech Today

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Magazine

Gene therapy

A Biotechnology designed to recreate life.

1.Background: Biotechnology

1.1 Definition of biotechnology and genetic engineering......4

1.2 Importance of disciplines in the scientific context...4

2.1 History......6

2.2 Types and applications......7,8

3. Content: Gene therapy

3.1 Gene therapy 1. Reprogramming life 2. How does it work? 3. Types and approaches 4. Applications...10

3.4 Genes involved......13

3.3 Key biomolecules......12

3.2 Organisms and cells used......11

4.1 Applied biological processes......15

4.From laboratory to society

4.2 Products Obtained......16

5.1 Advantages and disadvantages......18

5. Biotechnology under the microscope

5.2 Conclusions......19

2. Past, Present and Future of Biotechnology

ÍNDICE

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01

Biotechnology

Concepts and basic fundamentals

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BIOTECHNOLOGY: It is the application of living organisms and their processes to obtain products or services that benefit humans. In essence, it is the manipulation of biological systems for practical purposes.

GENETIC ENGINEERING: It is a branch of biotechnology that focuses on the direct manipulation of the genetic material of an organism. It allows genes to be modified, eliminated or introduced to obtain desired characteristics.

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02

Past, Present and Future of Biotechnology

A journey through time: Milestones and characters

XX CENTURY

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A journey through time

The discovery of the structure of DNA by watson and crick in 1953 marked a before and after. The pcr technique, developed by kary mullis, revolutionized genetic research.

THE SEQUENCING OF THE HUMAN GENOME AND THE DEVELOPMENT OF GENE-EDITING TECHNIQUES, SUCH AS CRISPR-CAS 9, HAVE OPENED UP NEW FRONTIERS IN BIOTECHNOLOGY.

XXI CENTUARY

Fermentation to produce beer, wine, and bread is one of the first applicacion of biotecnology.

Antiquity

Biotechnology is classified into diferent tipes according to its application:

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Red biotechnology: Focuses on human health, including the development of drugs, vaccines, and gene therapies.

White biotechnology: Applied to industrials processes, such as the production of biofuels, enzymes and bioplastics.’

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Green biotechnology: Related to agriculture, including crop improvement, biofertilizer production, and biodegradation of contaminated soils.

Blue biotechnology: Focuses on marine and aquatic organisms.

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03

Gene Therapy

Biotechnological Components & Mechanisms

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How does it work?

The process of gene therapy involves introducing a functional gene into a patient's cells. This is achieved through the use of vectors, which are vehicles that transport the therapeutic gene into the nucleus of the cell. The most common vectors are viruses, modified to be safe and efficient in delivering the genetic material. Once inside the cell, the therapeutic gene is incorporated into the genome and begins to produce the protein needed to correct the genetic deficiency.

Steem cells

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Bacterias

They are the most common vehicles for transporting therapeutic genes into cells. Their natural ability to infect cells makes them ideal tools for gene therapy.

They are used in the production of recombinant proteins, which can be used as drugs or to generate viral vectors.

These cells have the ability to differentiate into different cell types and are used in gene therapy to generate healthy tissues and replace damaged ones.

Viruses

Organisms and Cells Used

Proteins

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Viruse

Nucleic acids (DNA and RNA)

Key Biomolecules

• They are the undisputed protagonists. DNA contains the genetic information, while RNA is essential for gene expression. In gene therapy, DNA fragments encoding therapeutic genes are used.

• Proteins play diverse roles in the process, from the formation of viral vectors to the expression of therapeutic genes. Restriction enzymes and ligases are essential tools for manipulating DNA.

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Genes involved

The specific genes used in gene therapy vary according to the disease being treated. Some examples include:

Missing protein-coding genes

In diseases such as cystic fibrosis, a functional gene is introduced to replace the defective one.

Tumor suppressor genes

They are used to fight cancer by inhibiting uncontrolled cell growth.

Genes that stimulate the immune system

They are used to treat infectious diseases and cancer.

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04

From laboratory to society

Biotechnological Products and Processes

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Molecular cloning

Genetic recombination

Transformation

Transcription and translation

Applied Biological Processes

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They are used in research and in the production of substances of biomedical interest.

Used for transplantation and to produce therapeutic proteins.

Gene therapy has led to the development of new drugs to treat genetic diseases, cancer and other conditions.

Products Obtained

Gene Therapy

Medications

Genetically modified cells

Genetically modified organisms

Info

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05

Biotechnology under the microscope

Advantages, disadvantages and reflections

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advantages and disadvantages

ADVANTAGES

• Treatment of incurable diseases: Offers hope to patients with genetic diseases.
• Treatment can be tailored to the individual needs of each patient.
• Development of new therapies: Promotes research and development of new therapeutic strategies.

DISADVANTAGES

• The cost is very expensive.
• Side effects: There is a risk of unwanted side effects, such as immune reactions or insertion of the gene into an unwanted location in the genome.
• Ethical aspects: Modification of the human genome raises important ethical dilemmas.

• GENÉTICA
• TERAPIA
• ENFERMEDADES
• BIOMOLÉCULAS
• ADN
• ARN
• CLONACIÓN
• LUXTURNA
• YESCARTA
• ZOLGENSMA
• TECARTUS

Activity

COMPLETE THE LETTER SOUP

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conclusions

Written By : Anguiano Robles Karoll Arely

Written By: Contreras Monteñez Jazmin Guadalupe

Written By: Franco Carrillo Santiago

Written By: Juarez Carranza Luz Estrella

Written By: Lopez Ramirez Daniel Nicolas

Written By: Trejo Alcantara Emma Sofia

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• Advances in the industry: Production of biofuels, industrial enzymes, bioplastics and other biotechnological products.
• Environmental protection: Bioremediation of contaminants, development of crops tolerant to drought and salt stress, and production of biofertilizers.

Practical importance

Importance of both disciplines in the scientific and practical context

Scientific Importance

• innovation in medicine: Development of new drugs, gene therapies, disease diagnosis and vaccine production.
• creation of scientific tools: techniques such as CRISPR-CAS9, for gene editing; animals with genetic modifications for research

• Improvements in agriculture: Creation of crops more resistant to pests and diseases, increased food production and development of genetically modified crops.

A study was done where it was found that viruses such as adenoviruses, retroviruses and lentiviruses are genetically modified to eliminate their ability to cause disease and use them as vectors.

Nowadays, genetic engineering can become the safe future for new generations, since thanks to the research, developments and tools that have been used in the biotechnological era, DNA has been modified to suit the prevention of genetic diseases, which centuries ago was thought impossible, and has even given hope to those people with terminal illnesses with gene therapies, this giving more specialized attention to treatments with individual needs.Even so, it is important to remember that these new techniques do not exceed human ethics since it could be dangerous to see the consequences in future generations with genetic defects.

Gene therapy demonstrates a constantly evolving field where scientific advancements are continually increasing. As researchers delve deeper into the understanding of the human genome and develop new genetic editing tools, the possibilities of this area expand. However, it is important to remember that gene therapy is not a magic cure or solution to all existing ills or problems. Each disease presents its own challenges, and finding a cure for all of them will require a collaborative effort on a global scale. Gene therapy is, ultimately, a testament to human ingenuity and our ability to rewrite the book of life.

Some approved gene therapy products include

• LUXTURNA: Used to treat retinal dystrophy.

• YESCARTA: Used to treat diffuse large cell B lymphoma.

• ZOLGENSMA: Used to treat spinal muscular atrophy.

• TECARTUS: Used to treat mantle cell lymphoma.

The history

The history of biotechnology and genetic engineering is rich in discoveries and advances.

Biotechnology is the result of great advances and discoveries by implementing cells for different purposes, such that have helped the development of new drugs, transgenic products, biofuel, etc.; which have had a positive effect on society by making things easier to do both for human well-being and the environment. We must be aware that it can have consequences because the costs are high for different innovative procedures as well as ethical problems.

Results

In this way, diseases caused by a single defective gene, such as cystic fibrosis, hemophilia and some forms of cancer, can be treated.

Gene therapy is very productive and beneficial for humans and living beings, as it can cure hereditary diseases and some types of cancer which is very productive. But as well as it has its great contributions it can be very expensive or it can have great consequences such as receiving cancer and other reactions from your body. In my opinion it has a great future in terms of clinical development, but it may take too long, Our genes will have to wait.

Production of ethanol from biomass for use as fuel.

EXAMPLES

Production of bioactive compounds from algae.

EXAMPLE:

Gene therapy represented a 360-degree shift for the medical community and society at large. Although it still faces challenges such as high costs, technical complexity, and ethical considerations, advances in research are driving its safety, efficacy, and accessibility. As it continues to improve, gene therapy has the potential to transform the healthcare landscape, bringing hope to millions of patients worldwide. This is why I consider it extremely important for society as a whole to be aware of its existence and how it works. Only through proper understanding can we foster its acceptance, regulation, and responsible application. Moreover, an informed public will enable the ethical and social debate around gene therapy to be more inclusive, promoting fair and equitable development that benefits all of humanity.

Gene Therapy today is too great a tool. The thought of being able to cure a disease such as cancer is an opportunity that did not exist years ago. Even if this were to happen, it is clear that not all people will be able to access this service due to the economic differences that have always existed. Gene therapy came to show us that humans are capable of creating anything, they do all this for their own benefit and that is why, although we do not know if cancer or any other disease will ever be curable, it is admirable to know that with biotechnology it is possible to achieve it.

Insulin production for diabetic patients

EXAMPLES:

Development of herbicide-resistant crops.

EXAMPLE: