introduction to gene therapy a clinical aftermath

... biopsy-documented failure to respond to cyclophosphamide alone Scand J Rheumatol 2004, 33:423-427 Tokunaga M, Fujii K, Saito K, Nakayamada S, Tsujimura S, Nawata M, Tanaka Y: Down-regulation of CD40 and CD80 ... Rituximab administration was started The clinical response to Rituximab was evaluated by routine laboratory tests, and clinical examination at days 30, 60 and 90 Complete renal response was defined ... regulatory T cells in rheumatoid arthritis and reversal by anti-TNF-alpha therapy J Exp Med 2004, 200:277-285 18 Vigna-Perez M, Abud-Mendoza C, Portillo-Salazar H, AlvaradoSanchez B, Cuevas-Orta...

Ngày tải lên: 09/08/2014, 08:22

... Ottawa Apoptogen Incorporated Ottawa, Ontario, Canada Chapter 13 Yoko Yoshida, M.S Department of Molecular Biotherapy Research Cancer Chemotherapy Institute Cancer Institute Tokyo, Japan Chapter ... Cellulaire Génétique Virale Ecole Nationale Vétérinaire D'Alfort Maisons Alfort, France Chapter 17 Meizhen Feng, Ph.D Gene Therapy Program University of Alabama at Birmingham Birmingham, Alabama, ... Ottawa Apoptogen Incorporated Ottawa, Ontario, Canada Chapter 13 John J Rux, Ph.D The Wistar Institute Philadelphia, Pennsylvania, U.S .A Chapter Silvia Santamarina-Fojo, M.D., Ph.D Molecular...

Ngày tải lên: 10/04/2014, 10:58

... 900527 Constanta, Romania Dorin Andrica Faculty of Mathematics and Computer Science Babeş -Bolyai University Str Kogalniceanu 3400 Cluj-Napoca, Romania dandrica@math.ubbcluj.ro and King Saud University ... Alexandria was the center of mathematical learning The period from 250 bce to 350 ce in Alexandria is known as the Silver Age, also the Later Alexandrian Age This was a time when mathematicians were ... Andreescu Dorin Andrica Ion Cucurezeanu An Introduction E to Diophantine Equations A Problem-Based Approach Titu Andreescu School of Natural Sciences and Mathematics University of Texas at Dallas...

Ngày tải lên: 05/06/2014, 18:59

... limitation Immune responses to vector Genome integration Longterm expression Main Viral Vectors Features Retroviral Lentiviral Adenoviral AAV Hum Foamy Vir advantages gene transfer in gene transfer ... Characteristics of Gene Delivery Vehicles Viral Vectors Features Retroviral Lentiviral Adenoviral AAV Hum Foamy Vir Viral RNA RNA DNA DNA RNA Yes G1 phase No No No genome Cell division Viral Vectors Features ... modified autologous cells after gene transfer in the laboratory The latter approach offers opportunities to integrate gene transfer techniques with cellular therapies (Chap 67) Table 65-1 Characteristics...

Ngày tải lên: 07/07/2014, 01:20

... therapy trial for another form of SCID, adenosine deaminase (ADA) deficiency (Chap 310) ADA-SCID is clinically similar to X-linked SCID, although it can be treated by enzyme replacement therapy ... that insertional mutagenesis leading to cancer was more than a hypothetical possibility As a result of the experience in these trials, all protocols using integrating vectors in hematopoietic cells ... this approach is sufficiently safe to be routinely recommended as an alternative to PEG-ADA Other diseases likely to be amenable to transduction of HSCs include Wiskott-Aldrich syndrome (trials...

Ngày tải lên: 07/07/2014, 01:20

... sight has been restored after subretinal injection of an AAV vector expressing RPE65 Transgene expression appears to be stable, with the first animals treated >5 years ago continuing to manifest ... limitations imposed by age await clinical studies AAV-RPE65 trials have now been approved in both the United States and Great Britain Other inherited retinal degenerative disorders may also be amenable ... knockdown of VEGF Early-phase trials of siRNAs that target VEGF RNA are underway, but these require repeated intravitreal injection of the siRNAs; an AAV vector–mediated approach might allow long-term...

Ngày tải lên: 07/07/2014, 01:20

... by autologous transplantation, is being investigated as a strategy for allowing administration of higher doses of chemotherapy than would otherwise be tolerated Gene Therapy for Vascular Disease ... ischemia) or cardiac muscle (angina/myocardial ischemia) Initial treatment options for both of these groups include mechanical revascularization or medical management, but a subset of patients are ... presentation of tumor antigens These immunologic approaches may be of particular value in treating minimal residual disease after other anticancer modalities Gene transfer strategies have also been developed...

Ngày tải lên: 07/07/2014, 01:20

... increasingly important as a therapeutic modality in the twenty-first century A central question to be addressed is the long-term safety of gene transfer, and regulatory agencies have mandated a ... predominantly integrating [retroviral, lentiviral, herpesvirus (latency and reactivation)], or non-integrating (plasmid, adenoviral, AAV)? What was the route of administration of the vector? What was ... with genetically enhanced T lymphocytes Nat Rev Cancer 3:35, 2003 [PMID: 12509765] Shah PB, Losordo DW: Non-viral vectors for gene therapy: Clinical trials in cardiovascular disease Adv Genet...

Ngày tải lên: 07/07/2014, 01:20

... Altaha R, Abraham J: Paraneoplastic neurologic syndrome associated with occult breast cancer: a case report and review of literature Breast J 2003, 9:417-419 Osako T, Ito Y, Morimatsu A, Tada ... checked and was elevated at 360 IgG remained greater than 20 A CT scan was therefore performed demonstrating cervical and axillary lymphadenopathy There was no visceral disease Biopsy of the axillary ... Sakurai N, Takahashi S, Akiyama F, Iwase T, Hatake K: Flare-up of dermatomyositis along with recurrence of breast cancer Breast J 2007, 13:200-202 This lady's quality of life improved substantially...

Ngày tải lên: 09/08/2014, 07:22

... El-Zaatari FA, Naser SA, Sandoval J: Open clinical trial of rifabutin and clarithromycin therapy in Crohn's disease Dig Liver Dis 2002, 34(1):22-28 Hafner R, Bethel J, Power M, Landry B, Banach ... to a generalised inflammatory relapse, of which the uveitis was part of; this was not, however, clinically apparent during the acute presentation The protracted subsequent recovery is atypical, ... and ocular hypertension completely resolved and all topical medications were discontinued At this stage, the patient's inflammatory bowel disease was managed using azathioprine 50 mg twice daily,...

Ngày tải lên: 11/08/2014, 10:22

... RNAs have been used in vitro to target viral genes like tat and rev [31] and cellular genes like CCR5 [32] with great success Protein and nucleic acid-based strategies Aptamers In general, aptamers ... positive and had malignancies One had leukemia and the other had refractory Hodgkin's lymphoma Fourteen days prior to receiving gene therapy, the researchers stopped their cyclosporine and HAART medications ... medications Six days prior to gene therapy, the patients received fludarabine-cyclophosphamide containing regimen (non-myeloablative) for five days and then cyclosporine and HAART was restarted Each...

Ngày tải lên: 14/08/2014, 19:22

... 5'-gcccagctatgaactccttct-3' and 3'-tgaagaggtgagtggctgtct-5'; INDO: 5'-ggtcatggagatgtccgtaa-3' and 3'-accaatagagagaccaggaagaa-5'; GTSE1: 5'acgtgaacatggatgacccta-3' and 3'-gttcgggaaccggattattta-3'; ... CDC2: 5'-ggtcaagtggtagccatgaaa-3' and 3'-ccaggagggatagaatccaag-5'; and MKI67: 5'-ccccaaccaaaagaaagtctc-3' and 3'-gactaggagctggagggctta-5' Histopathology and immunohistochemistry on paraffinembedded ... 3'-ctggaaggtggacagcga-5'; LTB: 5'-gaggaggagccagaaacagat-3' and 3'-tagccgacgagacagtagagg-5'; CCL5: 5'-catattcctcggacaccacac-3' and 3'-gatgtactcccgaac- Page of 13 (page number not for citation purposes)...

Ngày tải lên: 09/08/2014, 14:20

... Spacek LA, Gray RH, Wawer MJ, Sewankambo NK, Serwadda D, Wabwire-Mangen F, Kiwanuka N, Kigozi G, Nalugoda F, Quinn TC: Clinical illness as a marker for initiation of HIV antiretroviral therapy ... World Health Organisation: Antiretroviral therapy for HIVinfections in adults and adolescents: recommendations for a public health approach 2006 World Health Organisation: Towards Universal Access: ... RA, Fontanet AL: Evaluation of the World Health Organization staging system for HIV infection and disease in Ethiopia: association between clinical stages and laboratory markers Aids 1999, 13(3):381-389...

Ngày tải lên: 10/08/2014, 05:21

... Shibata H, Toyama K, Shioya H, Ito M, Hirota M, Hasegawa S, Matsumoto H, Takano H, Akiyama T, Toyoshima K, Kanamura R, Kanegae Y, Saito I, Nakamura Y, Shiba K, Noda T Rapid colorectal adenoma formation ... prior to use Use of AAV Vectors for Gene Therapy A major advantageous characteristic of AAV vectors is their ability to transduce nondividing cells AAV vectors have been used to transfer genes into ... information into a target cell (c) Packaging Cells The AAV vector alone cannot produce an AAV particle because the rep and cap genes are not present These AAV genes need to be present in a packaging...

Ngày tải lên: 10/08/2014, 14:22

... techniques are advantageous for gene therapy approaches when applied to hematological diseases Aspects of ex vivo gene transfer as well as certain gene transfer vector systems are particularly useful ... attempted to translate the genetic observations, and in some cases molecular tricks, found to work in lower eukaryotes or bacteria into the mammalian cell targeting arena An early observation by yeast ... from Bacteria and Yeast The ability to find and accurately pair DNA molecules enables accurate gene targeting Biochemically, the overall process can be thought of as a series of steps in a reaction...

Ngày tải lên: 10/08/2014, 14:22

... observations have laid the basis for consideration of gene transfer as a therapeutic approach to cardiac disease Additionally, both adenoviral and adenoassociated viral vectors can be delivered to ... GENE THERAPY IN CARDIOVASCULAR DISEASE disease In addition, angiogenic gene therapy may offer an adjunct to traditional therapies that improves long-term outcomes GENE THERAPY OF VASCULAR GRAFTS ... Critical role of secretion signal in use of naked DNA Cardiovasc Res 35:470–479, 1997 Cardiac Gene Therapy Akhter SA, Skaer CA, Kypson AP Restoration of beta-adrenergic signaling in failing cardiac...

Ngày tải lên: 10/08/2014, 14:22

... Acid-Based Gene Therapy Strategies for Cancer Treatment Approach Structure Mechanism Target Antisense DNA or RNA Translation arrest RNase H activation mRNA-oncogenes Antigene DNA or RNA Triplex formation ... Gene Expression in Cancer Gene Therapya Cancer Cells Promotors Breast and Mammary carcinoma MMTV-LTR; WAP-NRE; b-casein; SLPI; DF3(MUC1); c-erbB2 Neuroblastoma and glioblastoma Calcineurin Aa; ... tumor suppressor genes The data generated to date indicate that in patients with advanced cancers that are refractory to conventional therapies, cancer gene therapy techniques may mediate tumor regression...

Ngày tải lên: 10/08/2014, 14:22

... hemophilia A and B) provide larger markets where gene therapy could have an impact As with any new therapy, gene therapy approach for a disease state would need to have advantages over treatments ... plasmid DNA for human clinical investigations To proceed to advanced clinical trials and ultimately gain regulatory approval, the pharmaceutical development of gene therapy products will have to ... control of reagents can lead to the cessation of clinical trails of gene therapy protocols (see Chapter 13) Laboratory Scale Purification As the clinical aspects of gene therapy continue to grow,...

Ngày tải lên: 10/08/2014, 14:22

... Regulatory authorities also have a role to play Clinical trials are based on extensive preclinical experimentation and animal trials that take many years to complete Clearly, the particular vector system ... virus was administered to animals viral replication per se did not appear to have an overt pathogenetic affect, rather a T-cell lymphoma eventuated [62], presumably as a result of oncogene activation ... X-SCID gene therapy trail certainly was The concern is that these events will now cause a significant backlash against the use of all retroviral vectors while the real message is that we need to make...

Ngày tải lên: 14/08/2014, 19:22