GENE THERAPY-pendahuluan
Download
Report
Transcript GENE THERAPY-pendahuluan
GENE THERAPY
AN INTRODUCTION
Agustina Setiawati, M.Sc., Apt
2 sks 12 kali pertemuan
USIP 40%, UAS 40%, Tugas/Kuis 20%
DEFINITION
Terapi gen merupakan teknik yang digunakan
untuk membenarkan gen yang rusak yang
menyebabkan terjadinya suatu penyakit
What’s gene therapy?
I.
Imagine that you
accidentally broke one
of your neighbor's
windows.
Stay silent: no one will ever find
out that you are guilty, but the
window doesn't get fixed.
II. Repair it with some tape: not the
best long-term solution.
III. Put in a new window: not only do
you solve the problem, but also
you do the honorable thing.
So, if a flawed gene caused our
"broken window," can you "fix" it?
What are your options?
.
I.
II.
III.
Stay silent: ignore the genetic
disorder and nothing gets fixed.
Try to treat the disorder with drugs
or other approaches: depending
on the disorder, treatment may or
may not be a good long-term
solution.
Put in a normal, functioning copy
of the gene: if you can do this, it
may solve the problem!
DOGMA CENTRAL
DNA EUKARIOT
Eukaryotic Transcription
Cytoplasm
DNA
Transcription
RNA
RNA
Processing
mRNA G
Nucleus
G
AAAAAA
Export
AAAAAA
Disease that was cured by Gene Therapy
10
11
Pendekatan Terapi Gen
Memasukkan gen normal pada lokasi non-spesifik
Menghapus fungsi gen abnormal terapi antisense
Memperbaiki fungsi gen abnormal melalui reverse-mutasi yg
selektif
What is reverse mutation?
Mutasi - mjd +
Mutant, inactive
+
+-
+
WT, active
Mutate + to +-
-+
WT, active
WT ? active
Same site revertant different site revertant
Prinsip Kerja Terapi Gen?
1.
Penelitian awal : menyisipkan gen normal ke
dalam genom utk menggantikan gen yg
rusak/abnormal
2.
Molekul pembawa (vektor) membawa gen
terapetik ke dalam sel target pasien.
3.
Vektor yg paling umum adalah VIRUS, yg dapat
memasukkan DNA/RNA ke dalam sel manusia
Keuntungan Terapi Gen
1. Memampukan manusia mempunyai anak tanpa
‘konsepsi’
2. Menyembuhkan penyakit berbahaya tanpa
menimbulkan rasa sakit.
3. Mencegah penyakit genetik berdasarkan sex/gender
4. Meningkatkan ‘penerimaan’ organ hasil transplantasi
Kelemahan terapy gen
Keamanan- resiko efek genetik detrimental
Berbahaya karena menimbulkan 'genetic
reductionism'
Type of Human Gene Therapy
1. Somatic Gene Therapy
In vivo & Ex vivo
2. Germline Gene Therapy
SOMATIC GENE THERAPY
“ Transfer material genetic to the body cells
except germline”
GERMLINE GENE THERAPY
“ Involve genetic modification of germ cells”
Any question?
THANK YOU