PDF电子书：分子疗法（2011）

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3 @1 \9 r: R& |: |6 gMolecular therapies
! L6 s3 `, [6 T5 H1 Q) ADr. Zoltán Balajthy, Dr. János Aradi, Dr. Zoltán Balajthy, Dr. Éva Csősz, Dr. Beáta Scholtz, Dr. István Szatmári, Dr. József Tőzsér, Dr. Tamás Varga (2011); a5 Y4 M6 [) q- R: a* w: v
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University of Debrecen
5 Q' g5 L: A6 G& |3 q* o8 `' i7 X  @Copyright © 2011 The project is funded by the European Union and co-financed by the European Social Fund., Manuscript completed: 17 November 2011# N, [+ W8 a" b0 }+ J

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Index
8 U' v" K+ g$ \$ q8 c1. 1. Functional Genomics" _( }3 }5 U1 q
1.1 Definitions7 q( e0 ?& J. V* ?( B9 Y3 t$ ]7 N- Z
1.2 About diseases! h! H% G. _' G, Y) f
1.3 Approaches to understanding disease mechanisms
8 K- x/ L; {# H) g+ r+ {1 E1.3.1 Gene expression is regulated in several basic ways
& H! I) @! @) \) K# h5 K- v1 n1.3.2 Microarrays: functional genomics in cancer research3 ]1 A9 ^; ?9 a" J* c
1.3.3 A Variety of Genetic Alterations Underlie Developmental Abnormalities and Disease
3 [# ]7 X# Q( F; |7 r1.3.4 Genomic microarrays8 t. D. Z3 }+ O& K$ q7 ]% x% F. i' V
2. 2. Recombinant proteins
; J3 i3 i- y% l. T; V  B) X0 d2.1 Overview: Protein pharmaceuticals
/ J8 z! `% G9 B2.2 Cell-free systems: In vitro transcription and translation; q4 C7 k& o9 R2 s. A" V& S' i
2.3 Recombinant protein expression in isolated cells (cell culture)) W5 _. J+ ?. F1 H6 {# G: u7 o
2.4 Non-prokaryotic expression systems- l4 e  x0 d! m) c2 y2 b/ i4 g4 ]
2.4.1 Cloning in Pichia pastoris
  n2 e2 i, h- f6 j1 u4 o2.4.2 Baculovirus mediated protein expression in insect cells+ p& k: ]8 t& k) Z
2.4.3 Mammalian expression systems1 \; N3 [4 I2 d) ?& f
2.5 Purification of recombinant proteins
. b. k+ T3 W+ E3. 3. Gene Therapy: Vectors and Strategies.3 ~- Y  v4 O! J
3.1 Vectors for Gene Therapy
8 x$ k: `9 @+ `9 v# @  }9 Y4 p3.2. Types of gene transfer, vectors for gene therapy
; Z0 |9 W" l, |6 w, b! ~$ L3.3. General gene therapy strategies$ R1 C) s; E* q
3.4. Human gene therapy
& W& i0 K& T/ w: S) C# i4. 4. Protein replacement therapies.
% G  U, ^* c9 \4 c& ]5. 5. Recombinant antibodies and the phage display technology
9 w) v) q* S: Z) @9 }% C* Z5.1. Introduction8 o. @- S9 [0 t+ O
5.1.1. The structure of antibodies and their production in the body
  R; P# L" z8 e: J( s$ a. D0 j5.1.2. Antigen-antibody binding
* w7 Y. F5 z. P1 w2 O( ~" C1 ]5.2. The production of therapeutic antibodies( r8 C: y# n& X" [( J$ U% x2 ]$ l% p
5.2.1. The production of antibodies in hybridoma cells.
0 D4 I5 b" D* J3 p' A5.2.2. Humanized antibodies1 [% J- V* m! x2 ?
5.2.3. Production of human antibodies$ N* u$ j/ ~. Q5 `. U6 P3 r
5.3. Generation of antibodies by phage display& C3 ^6 D# o! U" G: |
5.3.1. The phage display technology2 R7 ~" G) W0 V: Z- g
5.3.2. Generation of phage libraries
& u6 b8 l) Z- z2 K7 k6 F5.4. Administration of therapeutic antibodies
6 @, z3 ^3 S  o- Q( R- i7 p6. 6. Anti-cytokine Therapy (sepsis).
$ L* H/ p! r% j' `6.1. The consequences of developing inflammation
5 X" w' C# S9 T  V6.2. Development of Inflammatory Response: Synthesis of Lipid Mediators. I4 c7 I' f. b) e
6.3. Role of the Liver in Maintenance of Homeostasis: Acute Phase Response
7 C; U% ~: L1 i: _& a3 G1 U6.4. Time-Course of the Inflammatory Response During Sepsis
1 `( ~9 h0 g  `- ]1 E& t% ^% r7. 7. Animal models and transgenesis in biotechnology; y5 v8 f( R  j/ T/ e6 Y7 H
8. 8. Embryonic and adult stem cells in regenerative medicine I.
- f$ ?; l. @/ l( d5 a. T" P( c8.1 Embryonic stem cells" E& B: D0 b% N
8.2 Somatic cell reprogramming into pluripotent stem cells5 A: G: r9 z" f9 H( \7 z
8.3 Adult stem cells
9 V- R4 S, R7 j9 Z) l' M; N9. 9. Embryonic and adult stem cells in regenerative medicine II.
& O) {( S9 h. d6 N, |& g0 S5 L# I9.1 Pluripotent stem cells for regenerative medicine7 G/ G, z) Q+ n! R
9.2 Clinical application of stem cells
+ Q( u$ m+ h7 o+ O9 h9.3 Stem cell therapy to cure various diseases
- D* a; Z% w8 v7 `) v10. 10.        Cell Cycle and Cancer Therapy, p53 I.
- ]& g+ c  ^5 H# p. g: ]0 z10.1 Cell Cycle
0 N5 ]2 E( j+ o  e' Q10.2. Mitogenic Signaling in Eukaryotic Cell Controls the Rate of Cell Division3 S0 f" E- S- o) y2 V
10.3. Biochemical Events of Cell-cycle – in M Phase
$ {& M# J2 d# e6 t' P& _( f10.4. Protooncogenes
# y) J' p7 H3 Z! }, A" V/ }7 }10.5. ErbB/HER Receptors8 w( ~* j' d5 B, B1 \* m
10.6. Therapeutic targets
- P$ h, j3 \6 Y) b% U; V& `11. 11. Cell Cycle and Cancer Therapy, p53 II.  K6 v3 |4 \0 `2 N3 _& d  i
11.1. Tumor Suppressor Genes and p531 d9 _9 g) C7 Y1 p; W
11.2. Biochemical Pathways of Apoptosis and its Therapeutic Utilization. y" h9 e7 O2 \( Y" L. |5 g, a
12. 12. Gene Silencing Technologies.
8 q" p+ ~0 l4 L0 m4 ^+ U12.1 Introduction5 X1 ?: r* A/ X5 D2 s: B7 W1 _6 |
12.2 Action of antisense oligonucleotides
( g( j3 H9 w4 A6 ^$ p9 W12.3 Chemical modifications of gene silencing oligonucleotides; general considerations
0 F2 Y. U3 k& v12.4 Inhibition of transcription by triple helix forming oligonucleotides
# z- _# O" e( z& ]5 n& p9 F12.5 Gene silencing by ribozymes
: R$ N' B/ X/ j4 A+ ~. _( i% M12.6 Gene silencing with short RNA fragments1 B4 E) o# z1 W- ]
12.7 Important final note% ~/ V5 d% L  \" ?
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