W Introduction to Gene Therapy and Therapeutic Cloning

Question 1

Differentiation results from ?

Answer 1

differential gene

expression.

Question 2

What does the Nuclear transplant and cloning experiments show .?

Answer 2

differentiated cell nucleus can retain totipotency and have the ability to act like a zygote nucleus and stimulate the production of an entire organism.

Question 3

Dolly sheep camefrom?

Answer 3

3 mothers

Question 4

Example of mammals that have been cloned?

Answer 4

mice,

rats, cows, pigs, goats, cats and even a horse!

Question 5

How to make nerve cells from ESCs?

Answer 5

Treatment of ESCs with a derivative of vitamin A

causes them to form nerve cells.

Question 6

Meaning of somatic cell nuclear transfer (SCNT)?

Answer 6

Transfer the nucleus from a normal patient cell to an

enucleated egg.

Question 7

Ways to Obtain Pluripotent Stem Cells?

Answer 7

Emberyonic stem cells

Induced pluripotent stem cells

Question 8

What is NOT permitted of SCNT?

Answer 8

✓ Embryo development beyond 14 days
✓ Embryo implantation into a woman’s or animal’s
body
✓ Production of embryos by egg and sperm for
research

Question 9

Why SCNT: Not yet achieved for humans?

Answer 9

Due to inefficiency of NT, and limited availability of eggs (ethical issue).

Question 10

Alternatives of SCNT?

Answer 10

• Use adult stem cells.
• Convert ‘ordinary’ cells to cells having stem cell
properties.

Question 11

Where we can found Adult Stem Cells (ASC)?

Answer 11

Found in tissues such as brain, bone marrow, skin, intestine

(all tissues?) being responsible for their replenishment

Question 12

Cells isolated from human fat (gives us )?

Answer 12

removed by liposuction) were shown to develop into muscle, bone and cartilage

Question 13

What are the Limits of ASC Potential?

Answer 13

1-the environment of stem cells limits their ability to specialise
2-cell’s
environment determines what an adult stem cell will do.

Question 14

Ways of Converting ‘Ordinary’ Cells to stem cells ?

Answer 14

a -classical development and hESC derivation.

b-standard nuclear transfer

Question 15

How many genes coding in the Reprogramming Mouse Skin Cells

?

Answer 15

4 genes

Question 16

Rapid Advances Using iPS Cells .?

Answer 16

Successful treatment of mouse sickle cell anaemia by iPS from autologous skin (2007)
• Human iPS cells (2007-8)
• Creation of virus-free human iPS cells (2009) • iPS cells are generated from disease cells.
)
✓ 82 year-old patient with amyotrophic lateral sclerosis (ALS); made iPS
cells and directed them to form motor neurons the cell type destroyed
in ALS

Question 17

Why Recent Reports Call for Caution of ips?

Answer 17

Epigenetic differences

Genomic abnormalities:

Question 18

Which type of disease Human Gene Therapy (HGT) treat ?

Answer 18

homozygous for a recessive mutation
lacking a functional gene product.
inherited disease
but includes cancer and infectious diseases.

Question 19

Current gene therapy practice involves?

Answer 19

addition NOT correction or replacement.

Question 20

First Clinical Trial of Gene Therapy

Answer 20

1990
Ashanti )م adenosine
deaminase (ADA) deficiency.
infected with a normal ADA gene in retroviral vector.

Question 21

Cancer vector ?.

Answer 21

oncolytic adenovirus vectors

Question 22

First Trial For A Haemoglobin Disorder Using which vector ?

Answer 22

Lentiviral Vector

Question 23

Improvements in Gene Delivery are Still Needed.?.

Answer 23

• Specificity and improvement of gene transfer.
• Specificity, magnitude and duration of gene
expression.
• Avoiding stimulation of the immune system by some
vehicles.
• Manufacturing problems.
n.b. There is no ideal delivery vehicle for all applications.

Question 24

X-linked adrenoleukodystrophy (ALD) is a

neurodegenerative disease caused by?

Answer 24

mutation in the ABCD1 gene.