Gene Therapy
This book presents to the reader a comprehensive and detailed, yet easily accessible, description of the whole field of gene therapy. It covers topics ranging from vector development to the results of the most recent clinical trials. It includes descripti
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A α-fetoprotein (AFP) 197 α1-antitrypsin (AAT) 268 αvβ5, integrin 100, 111, 113 AADC cf. aromatic L-amino acid decarboxylase AAT cf. α1-antitrypsin AAV cf. adeno-associated virus AAVS1 116-118, 121 Ab-MLV cf. abelson murine leukemia virus ABC cf. ATP-binding cassette proteins Abelson murine leukemia virus (Ab-MLV) 67, 71 AC cf. adenylate cyclase ACE cf. angiotensin-converting enzyme Acetaldehyde 18 ACS cf. acute coronary syndrome Acquired immunodeficiency syndrome cf. human immunodeficiency virus Acute coronary syndrome (ACS) 230 ACV cf. acyclovir Acyclovir (ACV) 189, 190 AD cf. Alzheimer’s disease ADA cf. adenosine deaminase Adeno-associated virus (AAV) 3, 5-7, 40-42, 50-52, 112-122, 130-132, 141, 144, 170, 171, 176-180, 185, 186, 208, 213, 214, 216-220, 226, 228, 240, 241, 244, 245, 266, 268, 270, 288, 290 - genome organization 114 - replicative cycle 116 - structure of virions 113 Adeno-associated virus vectors 118-122 Adenosine deaminase (ADA) 2, 155, 156, 158 Adenovirus 5, 12, 39, 50-52, 63, 65, 95-104, 106, 108-110, 112, 113, 116, 118, 119, 132,
141, 142, 144, 176, 185, 191-193, 200-202, 239, 264, 269, 284 - function of adenoviral proteins 97 - genome organization 96 - replicative cycle 100 - structure of virions 95 Adenoviral vectors 102-112 - gutless 102, 103 - production 104-108 - properties 110 - purification 108 Adenylate cyclase (AC) 231, 243 Adrenoleukodystrophy 167 AEV cf. avian erythroblastosis virus AFP cf. α-fetoprotein Age-related macular degeneration (AMD) 34, 36, 144, 226 AIDS cf. human immunodeficiency virus AKT 231, 246 AlcR 19 Allogeneic, stem cell transplantation 147-149, 157, 159, 161, 165, 191 Alpharetroviruses 72, 79, 94 ALS cf. amyotrophic lateral sclerosis ALV cf. avian leukosis virus ALVAC 201 Alzheimer’s disease 11, 15, 35, 121, 144, 146, 204, 207-210 AMCV-29 cf. avian myelocytomatosis virus 29 AMD cf. age-related macular degeneration AMV cf. avian myeloblastosis virus Amyloid precursor protein (APP) 209 Amyotrophic lateral sclerosis (ALS) 12, 35, 143, 204, 207, 208, 216-219 Ang1 cfr. angiopoietin-1 Angioblasts 234
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Angiogenesis 12, 31, 35, 54, 132, 142, 144, 146, 172, 193, 195, 227, 228, 230, 231, 233-237, 241 Angioplasty 26, 36, 231, 232, 239, 246-249, 284 Angiopoietin-1 235 Angiotensin-converting enzyme (ACE) 230 Antagomir 35, 38 Antennapedia 26 Antibodies 4, 6, 7, 10, 13-15, 37, 49, 63, 78, 95, 110, 111, 148, 159, 161, 184, 186, 188, 189, 194-196, 199, 200, 228, 250, 252, 254, 258, 259, 262, 264 - idiotype 196 - intracellular antibodies 10, 13-15, 63, 188, 189, 194, 196, 252, 254, 258, 262 - minibodies 13, 14 - scFv 13, 14, 63, 258 - structure 13, 15 Antigen presentation 98, 197, 199 Antigen presenting cell (APC) 17, 52, 53, 146, 195 Antisense 4, 6, 10, 21-23, 29, 31, 35, 38, 40, 41, 141, 145, 151, 178, 180, 188, 189, 208, 221, 228, 231, 248, 252, 254-257, 260, 262, 263 - oligonucleotide (ASO) 6, 22, 23, 145, 151, 178-180, 188, 189, 208, 220, 228, 231, 248 - RNA 4, 5, 10, 21, 35, 38, 40, 41, 252, 254256, 260 APC cf. antigen presenting cell Apheresis 147, 159 ApoB cf. a
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