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Precise Gene Editing In Monkeys Paves The
Way For Valuable Human Disease Models
AND
New Study Shows Promise For Preventing
Therapy Resistance In Tumor Cells
Vanessa Pérez Carrillo
Medicine student
III semester
Molecular biology
INTRODUCTION
Molecular biology
techniques have
become a really
important tool,
because scientists can
explore the humans´s
DNA and can modify
some charateristcs.

This important
investigation open the
panorama of what is
cancer and how
physians can access for
a better possible cure.
Precise Gene Editing In Monkeys
Paves The Way For Valuable Human
Disease Models
SCIENCEDAILY, JANUARY 30, 2014
 Thanks to the implement of CRISPR/Cas9
system, the investigators
have had the
posibility to modificate monkey’s genes , the
reason is
because targeted genetic
modification in monkeys is invaluable for the
generation of human disease models.

 the CRISPR/Cas9 system enables simultaneous
disruption of two target genes in one step
without producing off-target mutations.
Precise Gene Editing In Monkeys
Paves The Way For Valuable Human
Disease Models
SCIENCEDAILY, JANUARY 30, 2014
The CRISPR/Cas9 system is a gene
editing tool capable of targeting
specific DNA sequences in the
genome.
Cas9 proteins, which are directed by
molecules called single-guide RNAs
to specific sites in the genome,
generate mutations by introducing
double-stranded DNA breaks
Precise Gene Editing In Monkeys
Paves The Way For Valuable Human
Disease Models
The scientist injected messenger RNA
molecules encoding Cas9, in addition
to single-guide RNAs designed to
target three specific genes, into onecell-stage embryos of cynomolgus
monkeys.
After sequencing genomic DNA from
15 embryos, they found that eight of
these embryos showed evidence of
simultaneous mutations in two of the
target genes.
Precise Gene Editing In Monkeys
Paves The Way For Valuable Human
Disease Models
The
researchers
transferred
genetically modified embryos into
surrogate females, one of which
gave birth to a set of twins. By
sequencing the twins' genomic
DNA, they found mutations in two
of the target genes.

the CRISPR/Cas9 system did not
produce mutations at genomic
sites that were not targeted,
suggesting that the tool will not
cause undesirable effects when
applied to monkeys
OBSERVATION

I found this news really great because it shows me how the
medicine keeps improving everyday, how to search about new
alternatives for the study about our DNA, also let us know the
importance of some animals that have similitudes to humans for
evaluate disease models, compare the DNA and try to create and
discover new alternatives to make a correct prevention,
diagnosis and treatment of a lot of pathologies.
New Study Shows Promise For
Preventing Therapy Resistance In
Tumor Cells
JA
researchers
suggests
that
activating
the
tumor
suppressor p53 in normal cells
causes them to secrete Par-4.
Par-4 is another potent tumor
suppressor protein that induces
cell death in cancer cells.
New Study Shows Promise For
Preventing Therapy Resistance In
Tumor Cells
Loss of the tumor suppressor p53
often contributes to therapy
resistance in tumors.
The investigators activated wild
type p53 in normal cells to trigger
cell death in the p53-deficient
cancer cells.
p53 is intact and functional in
normal cells, the researchers
harnessed its potential to inhibit the
growth of p53-deficient cancer cells.
New Study Shows Promise For
Preventing Therapy Resistance In
Tumor Cells
The tumor suppressor Par-4
specifically kills cancer cells.
Par-4 is really special because it
is not mutated as frequently as
other known suppressors.
The secretion from normal cells
can be induced by activating p53
so that Par-4 enters circulation,
thereby potentially targeting
tumor cells at distant sites.
New Study Shows Promise For
Preventing Therapy Resistance In
Tumor Cells
Scientists gets to empower the
normal cells to trigger cell death in
p53-deficient cancer cells

All of this findings have potential for
targeting local, as well as metastatic
tumors, and future studies will use
FDA-approved drugs to induce Par-4
secretion.
OBSERVATION

I really like this news because I learned about the effects of
tumor suppressor Par-4, it shows us the way to implement a new
technique that let us know more about the tumor cell activity
and control the cell born and death. Its really usefull for
investigate the way of making studies to find solutions for
cancer. Also it invite me to be more interested in what can I do
for the humanity.
MEDICAL UTILITY 1

The knowledge about
primate´s genes is an
excellent opportunity for
science to research and
evaluate similitudes with
humans genes, and then, with
this nformation we can work
for a healthier life.
MEDICAL UTILITY 1

The implement of this
important, safer and specific
system CRISPR/Cas9 will
significantly advance the
development of therapeutic
strategies in biomedical
research
MEDICAL UTILITY 2

It give us a major source of
discovery and development of
more effective approaches to
cancer prevention, diagnosis
and treatment
MEDICAL UTILITY 2

It is really important to know
about the effects of the Par-4
protein because it is a special
way to find the possible cure
of cancer thanks to all the
qualities.
Bibliography

Martínez S. Lina María. Biología molecular, 5 ed. Medellín, UPB, facultad de
medicina.
Ravshan Burikhanov, Tripiti Shrestha, Nikhil Hebbar, Gerard P. Zambetti, Vivek
M. RangnekaR. Paracrine apoptotic effect of p53 mediated by tumor supresor
Par
4.
Cell
Report,
January
9,
2014.
(online
magazine)
http://www.sciencedaily.com/releases/2014/01/140109175519.htm
Yuju Niu, Bin Shen, Yiqiang Cui, Yongchang Chen, Jianying Wang, Lei Wang, Yu
Kang, Xiaoyang Zhao, Wei Si, Wei Li et al. Generation of Gene-Modified
Cynomolgus Monkey via Cas9/RNA- Mediated Gene Targeting in One- Cell.
Embryos.
Cell
30
January
2014.
(online
magazine)
http://www.sciencedaily.com/releases/2014/01/140130121607.htm
Folding

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Folding

  • 1. Precise Gene Editing In Monkeys Paves The Way For Valuable Human Disease Models AND New Study Shows Promise For Preventing Therapy Resistance In Tumor Cells Vanessa Pérez Carrillo Medicine student III semester Molecular biology
  • 2.
  • 3.
  • 4. INTRODUCTION Molecular biology techniques have become a really important tool, because scientists can explore the humans´s DNA and can modify some charateristcs. This important investigation open the panorama of what is cancer and how physians can access for a better possible cure.
  • 5. Precise Gene Editing In Monkeys Paves The Way For Valuable Human Disease Models SCIENCEDAILY, JANUARY 30, 2014  Thanks to the implement of CRISPR/Cas9 system, the investigators have had the posibility to modificate monkey’s genes , the reason is because targeted genetic modification in monkeys is invaluable for the generation of human disease models.  the CRISPR/Cas9 system enables simultaneous disruption of two target genes in one step without producing off-target mutations.
  • 6. Precise Gene Editing In Monkeys Paves The Way For Valuable Human Disease Models SCIENCEDAILY, JANUARY 30, 2014 The CRISPR/Cas9 system is a gene editing tool capable of targeting specific DNA sequences in the genome. Cas9 proteins, which are directed by molecules called single-guide RNAs to specific sites in the genome, generate mutations by introducing double-stranded DNA breaks
  • 7. Precise Gene Editing In Monkeys Paves The Way For Valuable Human Disease Models The scientist injected messenger RNA molecules encoding Cas9, in addition to single-guide RNAs designed to target three specific genes, into onecell-stage embryos of cynomolgus monkeys. After sequencing genomic DNA from 15 embryos, they found that eight of these embryos showed evidence of simultaneous mutations in two of the target genes.
  • 8. Precise Gene Editing In Monkeys Paves The Way For Valuable Human Disease Models The researchers transferred genetically modified embryos into surrogate females, one of which gave birth to a set of twins. By sequencing the twins' genomic DNA, they found mutations in two of the target genes. the CRISPR/Cas9 system did not produce mutations at genomic sites that were not targeted, suggesting that the tool will not cause undesirable effects when applied to monkeys
  • 9. OBSERVATION I found this news really great because it shows me how the medicine keeps improving everyday, how to search about new alternatives for the study about our DNA, also let us know the importance of some animals that have similitudes to humans for evaluate disease models, compare the DNA and try to create and discover new alternatives to make a correct prevention, diagnosis and treatment of a lot of pathologies.
  • 10. New Study Shows Promise For Preventing Therapy Resistance In Tumor Cells JA researchers suggests that activating the tumor suppressor p53 in normal cells causes them to secrete Par-4. Par-4 is another potent tumor suppressor protein that induces cell death in cancer cells.
  • 11. New Study Shows Promise For Preventing Therapy Resistance In Tumor Cells Loss of the tumor suppressor p53 often contributes to therapy resistance in tumors. The investigators activated wild type p53 in normal cells to trigger cell death in the p53-deficient cancer cells. p53 is intact and functional in normal cells, the researchers harnessed its potential to inhibit the growth of p53-deficient cancer cells.
  • 12. New Study Shows Promise For Preventing Therapy Resistance In Tumor Cells The tumor suppressor Par-4 specifically kills cancer cells. Par-4 is really special because it is not mutated as frequently as other known suppressors. The secretion from normal cells can be induced by activating p53 so that Par-4 enters circulation, thereby potentially targeting tumor cells at distant sites.
  • 13. New Study Shows Promise For Preventing Therapy Resistance In Tumor Cells Scientists gets to empower the normal cells to trigger cell death in p53-deficient cancer cells All of this findings have potential for targeting local, as well as metastatic tumors, and future studies will use FDA-approved drugs to induce Par-4 secretion.
  • 14. OBSERVATION I really like this news because I learned about the effects of tumor suppressor Par-4, it shows us the way to implement a new technique that let us know more about the tumor cell activity and control the cell born and death. Its really usefull for investigate the way of making studies to find solutions for cancer. Also it invite me to be more interested in what can I do for the humanity.
  • 15. MEDICAL UTILITY 1 The knowledge about primate´s genes is an excellent opportunity for science to research and evaluate similitudes with humans genes, and then, with this nformation we can work for a healthier life.
  • 16. MEDICAL UTILITY 1 The implement of this important, safer and specific system CRISPR/Cas9 will significantly advance the development of therapeutic strategies in biomedical research
  • 17. MEDICAL UTILITY 2 It give us a major source of discovery and development of more effective approaches to cancer prevention, diagnosis and treatment
  • 18. MEDICAL UTILITY 2 It is really important to know about the effects of the Par-4 protein because it is a special way to find the possible cure of cancer thanks to all the qualities.
  • 19. Bibliography Martínez S. Lina María. Biología molecular, 5 ed. Medellín, UPB, facultad de medicina. Ravshan Burikhanov, Tripiti Shrestha, Nikhil Hebbar, Gerard P. Zambetti, Vivek M. RangnekaR. Paracrine apoptotic effect of p53 mediated by tumor supresor Par 4. Cell Report, January 9, 2014. (online magazine) http://www.sciencedaily.com/releases/2014/01/140109175519.htm Yuju Niu, Bin Shen, Yiqiang Cui, Yongchang Chen, Jianying Wang, Lei Wang, Yu Kang, Xiaoyang Zhao, Wei Si, Wei Li et al. Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA- Mediated Gene Targeting in One- Cell. Embryos. Cell 30 January 2014. (online magazine) http://www.sciencedaily.com/releases/2014/01/140130121607.htm