Bioinformatics
Topic: Invitro, Invitro tools, Gene therapy, Gene design, Gene
Engineering, Steps for Re-Modeling of a Gene
Assignment No: 3rd
Submitted By: Asad Yar
Submitted To: Malik Mughees
Registration No: FA21-BCS-212
Date of Submission: Dec 29,
2022
Campus: Abbottabad
Introduction
In vitro is Latin for “within the glass.” When something is performed in vitro,
it happens outside of a living organism.
Invitro
You may have encountered the terms “in vitro” and “in vivo” while reading
about scientific studies. Or perhaps you’re familiar with them through
hearing about procedures such as in vitro fertilization. But what do these
terms actually mean? Continue reading as we break down the differences
between these terms, give some real-life examples, and discuss their pros
and cons.
Real-life Example
Now that we’ve defined these terms, let’s explore some real-life examples
of them.
Studies
In vitro, in vivo, or in situ methods are used in scientific studies. In some
cases, researchers may use multiple methods to test their hypothesis.
In-Vitro
In vitro methods used in a laboratory can often include things like studying
bacterial, animal, or human cells in culture. Although this can provide a
controlled environment for an experiment, it occurs outside of a living
organism and results must be considered carefully.
In-Vivo vivo
when a study is performed in vivo, it can include things like performing
experiments in an animal model, or in a clinical trail in the case of humans.
In this case, the work is taking.
In situ
In situ means “in its original place.” It lies somewhere between in vivo and
in vitro. Something that’s performed in situ means that it’s observed in its
natural context, but outside of a living organism.
Gene Therapy
Human gene therapy seeks to modify or manipulate the
expression of a gene or to alter the biological properties of living
cells for therapeutic use. Gene therapy is a technique that
modifies a person’s genes to treat or cure disease. Gene
therapies can work by several mechanisms:
Replacing a disease-causing gene with a healthy copy of the
gene
Inactivating a disease-causing gene that is not functioning
properly
Introducing a new or modified gene into the body to help
treat a disease
Gene therapy products are being studied to treat diseases
including cancer, genetic diseases, and infectious diseases.
There are a variety of types of gene therapy products, including:
Plasmid DNA: Circular DNA molecules can be genetically
engineered to carry therapeutic genes into human cells.
Viral vectors: Viruses have a natural ability to deliver
genetic material into cells, and therefore some gene therapy
products are derived from viruses. Once viruses have been
modified to remove their ability to cause infectious disease,
these modified viruses can be used as vectors (vehicles) to
carry therapeutic genes into human cells.
Bacterial vectors: Bacteria can be modified to prevent
them from causing infectious disease and then used as
vectors (vehicles) to carry therapeutic genes into human
tissues.
Human gene editing technology: The goals of gene
editing are to disrupt harmful genes or to repair mutated
genes.
Patient-derived cellular gene therapy products: Cells
are removed from the patient, genetically modified (often
using a viral vector) and then returned to the patient.
Gene therapy products are biological products regulated by the
FDA’s Center for Biologics Evaluation and Research (CBER).
Clinical studies in humans require the submission of an
investigational new drug application (IND) prior to initiating
clinical studies in the United States. Marketing a gene therapy
product requires submission and approval of a biologics license
application (BLA).
Gene Design
Gene design relies upon another major discovery. This was the
'One gene One
Enzyme' Theory first proposed by George W. Beadle and Edward L. Tatum
in the 1940's. Discoveries made during their research laid the groundwork
for the theory that a single gene stores the information that directs the cell
in how to produce a single enzyme.
Protein). Therefore, there is a single gene that controls the production of
the Bt protein. It is called the Bt gene.
Once a gene has been cloned, genetic engineers begin the third step,
designing the gene to work once inside a different organism. This is done in
a test tube by cutting the gene apart with enzymes and replacing certain
regions.
Gene Engineering
Genetic engineering (also called genetic modification) is a process that
uses laboratory-based technologies to alter the DNA makeup of an
organism. This may involve changing a single base pair (A-T or C-G),
deleting a region of DNA or adding a new segment of DNA. For example,
genetic engineering may involve adding a gene from one species to an
organism from a different species to produce a desired trait. Used in
research and industry, genetic engineering has been applied to the
production of cancer therapies, brewing yeasts, genetically modified plants
and livestock, and more.
Narration
Genetic engineering. Genetic engineering has changed over the years,
from cloning for analysis and laboratory use to truly synthetic biology for
understanding and new biomedical capabilities.
Steps of Re-Modeling of a Gene
There are several steps involved in the process of remodeling a gene:
Identify the gene that needs to be remodeled: This involves
identifying the specific gene that needs to be modified or altered in
some way.
Determine the desired change: The next step is to determine what
specific change or modification is desired in the gene. This could
involve altering the sequence of the gene, deleting or adding specific
sections of the gene, or expressing the gene at different levels.
Design the modification: Once the desired change has been
identified, the next step is to design the specific modification that will
be made to the gene. This may involve using computer algorithms to
design the modification, or using existing techniques such as
CRISPR/Cas9 to target specific sections of the gene.
Create a model system: In order to test and evaluate the gene
modification, it is often necessary to create a model system in which
the modified gene can be studied. This may involve using a cell line,
an animal model, or a plant model.
Implement the modification: Once the modification has been
designed and a suitable model system has been identified, the next
step is to actually implement the modification. This may involve using
techniques such as CRISPR/Cas9 to make the modification, or using
other techniques such as transfection or transformation to introduce
the modified gene into the model system.
Evaluate the effects of the modification: The final step is to evaluate
the effects of the modification on the model system. This may involve
analyzing changes in the expression or activity of the gene, or
studying the effects of the modification on the overall function or
phenotype of the model system.
Search Tool
Search allows you to search content created by tools within a site. It
achieves this by creating an index of all content that other tools make
available to the search tool. This index is updated automatically, so
as content is added to the site, it will be indexed.
For example, if the site has an Email Archive, as emails are posted to
the list, the content of those emails will be indexed and become
searchable in the search tool. Or, if the site has documents uploaded
to Resources, the documents will become searchable.
The Search tool indexes many different types of content, including
messages; web pages in HTML format; documents in DOC, PDF, or
RTF format; spreadsheets in XLS format; and Wiki pages.
