Gene Teraphy & Gene Slicing

GENE THERAPY

Replacing an abnormal gene with a healthy copy of the gene

GENE THERAPY

Inactivating an abnormal gene that is functioning improperly

Gene Expression

Process by which the instructions in our DNA are converted into a functional product, such as a protein.

mRNA & Protein

Gene is expressed when there is:

DNA MICROARRAY

is used to identify or determine gene expression and also regulation.

mutation

If all genes are turned ON, there is a possibility for ________ cell.

Insulin

Melanin

What is enzymes is off in the Muscle

Myosis

Melanin

What is enzymes is off in the pancreatic islet

DNA MICROARRAY

measure the expression of levels of large numbers of genes simultaneously or to genotype multiple regions of a genome.

biopsy sample

sample to get a suspected sample of healthy and cancer cells

Poly-T primers

Acts as “wire or adaptor” to the mRNA

Reverse transcriptase

An enzyme which will synthesize a strand of complementary DNA (cDNA)

green

color that indicates healthy cells

RED

color that indicates cancerous cells

YELLOW

color that indicates both healthy and cancerous cells

GENETIC ENGINEERING

Also known as recombinant DNA technology

GENETIC ENGINEERING

Comprises multiple techniques for the intentional manipulation of genetic material to alter, repair, or enhance form or function

GENE THERAPY

Experimental technique that uses genes to treat or prevent disease

1932

year when Genetic Engineering was first presented at Sixth International Congress of Genetics

1944

Macleod and McCarty

year and who suggested that genes could be transferred within nucleic acids

Clyde E. Keeler

first use the term gene therapy

1960s and 1970s

year that set the basis of molecular genetics and gene transfer

1970

Stanfield Rogers

at what year that The first deliberate transfer of foreign genes into human recipients with a therapeutic purpose, and was performed by ___________

Martin Cline

in july 1980, who headed another highly controversial human trial designed to treat B-thalassemia

1983

in this year, Enzyme-producing-gene-corrected cells were further theorized as viable approach for treating Lesch-Nyhan disease

Somatic Cell Gene Therapy

Will not be inherited in later generations

Germ Line Cell Gene Therapy

Example: genes are introduced to egg cell or sperm cell

Somatic Cell Gene Therapy

introduction of genes into bone marrow cells, blood cells,

Somatic Cell Gene Therapy

Can be Ex vivo or in vivo

In vitro gene therapy

Non-viral delivery system

Viral delivery system

In vitro gene therapy

Viruses currently being used for HCC gene therapy

Ex vivo gene therapy

Cells modified in vitro Modified cell – place inside the sterile petri dish

VIRAL VECTORS

Vehicle for gene delivery

Retrovirus

Adenovirus

Adeno-associated virus

Herpes simplex virus

TYPES OF VIRAL VECTORS

naked DNA

It is the simplest non-viral method

Naked DNA

Clinical trials carried out intramuscular injection of the _______ plasmid have occurred with some successes.

OLIGONUCLEOTIDES

Used of the synthetic oligonucleotides and the gene therapy can activate genes involved disease process

LIPOPLEXES AND POLYPLEXES

Improve the delivery of new DNA into the cell.

gene silencing

It generally describe the “switching off’ of a gene by a mechanism other than genetic modification

gene silencing

same as gene knock down but is totally different from gene knock out

Gene Silencing

Technique that aims to reduce or eliminate the production of a protein to its corresponding gene

Jorgesen

in 1990, who deepen the pigmentation in petunias Introduction of transgenes homologous to endogenous genes often resulted in plants with both gene suppressed called – co suppression

1995

In this year, Guo and Kemphues - Injection of either antisense or sense RNA in the germline of C.elegans was equally effective at silencing at homologous target genes

Craig Mello

Andrew Fire

in 1998, this two scientist provide an extension of the experiment of Guo and Kemphues

2001

Year when Synthetic exogenous siRNA to induce sequence-specific gene knockdown in mammalian cells in vitro

1998

year when the First description of dsRNA as trigger for gene specific silencing and term as RNAi

mouse

in year 2002, first in vitro gene slicing with RNAi is done in what animal?

2006

Year when a Systemic administration of siRNA in non-human primates. it is also the same year when Andry Fire and Craig awarded the Nobel Prize for discovery of RNAi

2008

Proof of concept for use of an RNAi therapeutic in humans was provided in this year

2013

This year provided Explosive growth for basic research and therapeutic intervention in various diseases