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Raushan Kumar Ranu BT/05/11

Introduction :


PowerPoint Presentation:

Change the regulation of gene pairs

The Beginning…:

The Beginning…

The First Case:

The First Case

PowerPoint Presentation:

Gene therapy Classical gene therapy Non-classical gene therapy

PowerPoint Presentation:

Classical gene therapy Non-classical gene therapy

Gene Therapy:

Gene Therapy Somatic cell gene therapy Germ line gene therapy

Germ line gene therapy:

Germ line gene therapy Somatic cell gene therapy

How It Works:

How It Works A vector is a carrier molecule, usually a virus The target cells are usually in the liver or lung.



Gene delivery system:

Gene delivery system 1.Virus mediated delivery method 2.Non-viraltransfection methods

1.Virus mediated delivery method:

1.Virus mediated delivery method This genetic material contains instructions to produce these viruses. The host cell will carry out these instructions and produce the viruses. In addition to the instructions producing the components of the virus itself , viruses can carry additional genes containing instructions for creating other kinds of proteins. Viruses attack their host to insert their genetic material into genetic material of host. Three types of viruses are currently used as vector in gene therapy.


Adenoviruses Adenoviruses are DNA viruses with a linear , double standed genome of apprx 36 kb. After insertion of genome of adenoviruses are not incorporated into genetic material of host cell. The linear dsDNA molecule are transcribed just like other gene .these extra gene also not replicates when the cell is about to undergo the cell division. So , the descendants of that cell will not have extra gene . This means that treatment with the adenovirus will require regular doses to add the missing gene every time. Advantages – can contain >30 kb of normal DNA , very high titer stocks , infects both dividing and non-dividing cells. Disadvantages - doesn’t provide long term gene expression due to lack of integration , very immunogenic.


Retroviruses The genetic material is RNA molecules. While the genetic material of their hosts is in the form of DNA. When a retrovirus infects a host cell , it will introduce its RNA together with some enzymes into the cell. This RNA molecule from the retrovirus must produce a DNA copy from its RNA molecule before it can be considered part of the genetic material of the host cell. The process of producing DNA copy from RNA called reverse transcription. Advantages – integrates into host cell genome providing stable gene expression , contain no viral gene . Disadvantages – infect only dividing cells , random integration may cause insertional mutations , relatively low titer stocks.

Adenovirus cont.:

Adenovirus cont.

Adeno-associated Viruses:

Adeno -associated Viruses

2.Non-viraltransfection methods:

2.Non-viraltransfection methods Direct introduction of therapeutic DNA - But only with certain tissue - Requires a lot of DNA Creation of artificial lipid sphere with aqueous core, liposome Carries therapeutic DNA through membrane . Chemically linking DNA to molecule that will bind to special cell receptors - DNA is engulfed by cell membrane - Less effective . Currently two methods are used – Liposomes mediated Biolistic method

Most common approaches-:

Most common approaches-

Problems with Gene Therapy:

Problems with Gene Therapy

PowerPoint Presentation:

Disease Target cell Transfected gene(s) Hemophilia - A Liver , Muscle , bone marrow cells Factor- VIII Hemophilia - B Fibroblasts Factor- IX Cystic fibrosis Lung airway cells Cystic Fibrosis Gene(CFTR) Gaucher’s disease Bone marrow cells, macrophages Glucocerebrosidase cancer Tumor cells p-53, interleukins , growth inhibitory gene , apoptosis genes .

Recent Developments:

Recent Developments

References :

References Biotechnology by B.D. Singh Biotechnology by Darbeshwar Roy Biotechnology by Pranav Kumar & Usha Mina

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