Name: Ayesha Siddique

ID: F2016231023

Subject: Applications of
Biotechnology

Assignment: 1
Gene therapy:-
Gene therapy is a clinical field which centers around the usage of the helpful conveyance of
nucleic corrosive into a patient's cells as a medication to treat malady. Gene therapy is an
approach to fix a genetic issue at its source. The polymers are either converted into proteins,
meddle with target gene articulation, or conceivably right changes. The most well-known
structure utilizes DNA that encodes a useful, helpful gene to supplant a changed gene. The
polymer atom is bundled inside a "vector", which conveys the particle inside cells.
There could be three possibilities to perform gene therapy.
1: Addition of a new gene in place of effected gene.
2: Removal of effected gene from the host.
3: Suppress the effected gene

Types of gene therapy

It is classified into two types:
 Somatic cell gene therapy
 Germline gene therapy

Germline gene therapy:-

In germline gene therapy, germ cells (sperm or egg cells) are altered by the presentation of
utilitarian genes into their genomes. Changing a germ cell makes all the life form's cells contain
the altered gene. The change is along these lines heritable and given to later generations.

Somatic cell gene therapy:-

In somatic cell gene therapy, the remedial genes are moved into any cell other than a gamete,
germ cell, gametocyte, or undifferentiated immature microorganism. Any such adjustments
influence the individual patient just, and are not acquired by posterity. Somatic gene therapy
speaks to standard essential and clinical research, in which restorative DNA is utilized to treat
sickness.

Methods of gene therapy

There are different methods for treatment of genetic disease by inserting genes so different
vectors can be used for transferring our desired gene in place of effected gene. Common studied
vectors are:
1. Adeno-Associated virus
2. Retrovirus
3. Adenovirus
 4. Non-viral vectors
 Retrovirus vector:-
These vectors are specifically used for dividing cells and need particular receptors for
proper functioning. There are some issues of using this type of vector & one of them is insertion
of a limited size of DNA.

 Non-viral vectors:-
Non-viral methods at first delivered lower levels of transfection and quality articulation,
and therefore lower helpful viability. Non-viral vectors can diminish the danger of
contaminations however the addition of qualities isn't so proficient. Another issue with
utilizing this vector is, it required restricted kind of cells. Methods for non-viral quality
treatment incorporate the infusion of

Exposed DNA, electroporation, the quality firearm, the utilization of oligonucleotides,

lipoplexes, dendrimers and in natural nanoparticles.
 Adeno-associated virus:-
Genes can be specifically inserted on specific sites by using adeno-associated virus
vectors. But if the insertion of genes will not be inserted on the focus point, it can cause
mutagenesis.
 Adenovirus vector:-
This type of vector is used for transferring genes in respiratory diseases in man. There is
no limitation of dividing cells, it can target different cells.

Problems:-
Stable expression:
Maintaining the expression at high level or for required time period can be a problem after gene
therapy.
Tissue-specific expression:
It could be the major issue to obtain an expression for specific tissues or organs which had the
effected genes in it.
In vivo regulation:
Drawbacks of in vivo gene move are the likelihood that the presentation and articulation of
infection proteins may actuate endogenous pathogenic infections. It has likewise been
recommended that replication-skilled recombinant infections may shape because of arbitrary
mutagenesis. The chance exists that infections, which incorporate themselves into the host
genome, for example, retroviruses, may cause unplanned harmful change of the host genome.
Aggravation and safe reaction are additionally of specific concern, even in an 'invulnerable
favored' organ, for example, the cerebrum. These dangers are expanded or diminished relying
upon the vector
Delivery of DNA:
Delivery of DNA on explicit site is the greatest issue in quality treatment. At some point outside
DNA can't reach on the ideal spot so these qualities or DNA appended and begins their capacity
on other spot which cause diverse harm to the cell or might be to the entire organs.
High level expression:
The statement of embedded DNA or quality can cause issue in light of the fact that occasionally
it doesn't show required degree of articulation which can treat the hereditary sickness. At some
point it shows articulation however the degree of articulation will be insufficient for the best
possible capacity of required qualities.