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Tze Huay
Jia Jing
Jia En
Jia Yii
Concept – arose during the 1960s and 1970s

A functional copy of the defective gene is introduced to replace the missing or incorrect function.

Development

1990 - First patient to be treated with gene therapy was a 4 y-ear-old girl treated for a congenital disease called Adenosine Deaminase (ADA) deficiency of which severely affects immunity and ability to fight infections.

Therapeutic Procedure ex vivo

1.     White blood cells of the patient were extracted

2.     The corrected human ADA gene was inserted into the cells via a retrovirus

3.     Corrected gene encapsulated in a retrovirus reinjected/reinfused back into the patient

4.     Repeated treatments conducted over 2 years

5.     Patients are now living normal lives with a steady increase in ADA in their immune cells

1972 - Theodore Friedmann and Richard Roblin published a paper in Science called "Gene therapy for human genetic disease?" which cited Stanfield Roger's proposal in 1970 that "good DNA" could be used to replace defective DNA in people with genetic disorders.

2012 - The first gene therapy was approved in the European Union

Types of gene therapy

Ex vivo gene therapy

Removal of genetically defective cells from patients à cells are grown in the laboratory and exposed to the virus carrying the desired gene à virus enters the cells and inserts desired gene into the cells’ DNA à corrected cells with desired DNA is injected/infused back into the patient.

In vivo and in vitro gene therapy

The corrected copy of genes is treated with a vector and are directly transferred into the body of the patient

-       Vectors may include:

o   Direct injection of recombinant virus (in vivo)

o   Use of biolistic gene guns for insertions of plasmid DNA (in vitro)

o   DNA liposome (in vitro)

In situ gene therapy

Vector encapsulated with normal gene are injected directly into patient’s abnormal tissue to be treated. This promote genetic alteration on the abnormal cells on the site of diseased tissue.

-       Effective against cystic fibrosis

Approved Applications:

T-cell immunotherapies

1. RPE65 mutation-associated retinal dystrophy – Luxturna Therapy

2. CAR T-cell therapy for acute lymphoblastic leukaemia and B-cell lymphoma (ALL)

3. Yescarta for large B-cell lymphoma (a type of non-Hodgkin lymphoma)

Developing Gene Therapies

1.     Genetic diseases (haematological, immunological, ocular, neurodegenerative, metabolic disorders)

2.     Eradication of blood cancers

3.     Correction of hemoglobinopathies

4.     Coagulation factor of deficiencies

5.     Restoration of immune system in children born with primary immune deficiency

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Wai Leong
Alicia
Theck Ruey

There are 2 categories of vaccines:

A)   Traditional vaccines:

1. Live-attenuated vaccines:

-        Live vaccines use a weakened (or attenuated) form of the germ that causes a disease

-        Example: BCG

2. Inactivated vaccines:

-        Inactivated vaccines use the killed version of the germ that causes a disease.

-        Example: V. cholera

3. Toxoid vaccines

-        Toxoid vaccines use a toxin (harmful product) made by the germ that causes a disease. They create immunity to the parts of the germ that cause a disease instead of the germ itself. That means the immune response is targeted to the toxin instead of the whole germ.

-        Example: DPT

B)    New vaccines

1. Subunit vaccines

-        A subunit vaccine presents an antigen to the immune system without introducing viral particles, whole or otherwise. One method of production involves isolation of a specific protein from a virus and administering this by itself.

2.  Vectored-based vaccines

-        Use of detoxified viral or bacterial host to carry the antigenic protein.

-        Example: Canarypox to carry envelope proteins for HIV

3.   DNA vaccines

-        DNA encoding the antigenic protein is injected. Cells that uptake these DNA will make the antigen to activate immune system.

4.  Peptide based vaccines

-        Chemically synthesized 8-24 amino acids antigenic peptides that mimic the epitopes to induce immune response.

-        Example: cancer vaccine

Influenza Flu (seasonal flu)

·       Caused by influenza virus type A, B, C

·       Undergo frequent mutation as they replicate

·       Antigenic shift in the hemagglutinin (H) and neuraminidase (N)

Influenza Vaccines

·       Killed/inactivated viruses or the antigenic envelope proteins

·       Specific to subtype and strain

·       Effective influenza vaccine requires an accurate prediction of the virus subtype and strains expected to circulate next flu season.

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Cytokine
Pui Yi
dikshita
Feerdauz

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P'ng
Haziq
Mohanad
Josiah

Traditional (polyclonal) antibodies
-immunoglobulins secreted by different B cell lineages
-identify different epitope

Monoclonal antibodies
-antibodies produced by identical immune cells
- identifying one specific epitope

Humanization of antibodies
-Humanized antibodies are antibodies from non-human species whose protein sequences have been modified to increase their similarity to antibody variants produced naturally in humans1. Chimeric antibodies consist of both murine(mouse antibodies) and human parts.
2. Humanized antibodies consist of mostly human antibodies and only specific antigen-binding region derived from mouse
3. Full human antibodies produced fully in transgenic mice with human antibody-producing genes.

-Antibodies produce in mice has limitation which will activates human immune system to produce anti-mouse antibodies and neutralize the effect of these antibodies and severe allergic reaction may occurs. therefore, development of mAb for administration to humans will humanized the antibodies.

Conjugated antibodies
-A conjugated antibody is a monoclonal or polyclonal antibody linked to a label(toxins or enzymes) and used for detection in a diverse range of assay techniques Ab binding and internalization into target cells ---> translocation of the toxin/fragment into cytosol / nucleus ---> localized action of compound.

Applications :-
Traditional(polyclonal) antibodies
- Injection of an antigen into an animal will result in a polyclonal antibody response in which different antibodies are produced that react with the various epitopes on the antigen. Polyclonal antisera are useful for some types of laboratory assays, but other assays require more specificity

Monoclonal antibodies
- utilized in diagnostic kits for diagnosis of various infectious disease, detecting pregnancy, monitoring  level, detect diabetes, detect cancer and much more.

Development:-Development :-
Monoclonal
- immunization of animals > harvest of antibody producing B-cells > formation of hybridomas > selection and culture of hybridoma production antigen specific antibodies.

Polyclonal
Immunization of animals> plasma collection(containing antibodies> antibody purification> formulation of antibodies into final preparation.

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Chan Le Roy
Siau Yaw Wen
Bilal Tahir

Development:
1) steroids : Synthesized from cholestrol in the gonads and andrenals, transported by bound carrier proteins - serum proteins e.g. Sex hormone - binding globulin.

2) Polypeptides: form by linkage of many amino acids through peptide bonds and modified.

3) Amino acid derivatives:

Types:
1) Steroids : lipid soluble ( easily pass through cell membrane) , 2 classes ( corticosteriod and sex steroids)
E.g estrogen and androgen
2) polypeptides: water soluble ( act on cell surface receptors and signal amplification via secod messenger), long amino chains
E.g Insulin
3) Amino acid derivatives :  water soluble ( same as polypeptides) , shorter amino acids chain
E.g. Adrenaline and Noradrenaline

Application:
1) Hormone Replacement Therapy (HRT) for menopausal women.
2) Androgen replacement therapy (ART) for men with low testosterone levels
3) Oncologic hormone therapy for cancer treatment