scholarly journals Overview of CFTR Modulators and Gene Therapy

2020 ◽  
Author(s):  
Catherine Rang ◽  
Tom Kotsimbos ◽  
John Wilson
Keyword(s):  
Gene Therapy ◽  
Cftr Modulators

Future treatments

2015 ◽  
Author(s):  
Rebecca Thursfield ◽  
Chris Orchard ◽  
Rosanna Featherstone ◽  
Jane C Davies
Keyword(s):  
Gene Therapy ◽  
Viral Gene ◽  
Viral Gene Therapy ◽  
Biofilm Disruption ◽  
Future Treatments ◽  
Clinically Meaningful Outcomes ◽  
Dose Trial ◽  
The Uk ◽  
Exciting Development ◽  
Cftr Modulators

There are only a relatively limited armoury of drugs, the majority of which are aimed at downstream symptoms of cystic fibrosis. Therapies targeting the basic defect in CF as well as continued availability of more conventional drugs are required. Progress in gene therapy has been limited by the significant barriers to gene transfer of the CF lung, but the UK is hosting a large repeated dose trial of nebulized non-viral gene therapy designed around clinically meaningful outcomes. The UK CF Gene Therapy Consortium is also seeking to develop a promising modified lentiviral approach, although this is some years off. Perhaps the exciting development of recent decades has come from small molecule CFTR modulators, driven by an understanding of basic pathophysiological mechanisms. Ivacaftor is the first drug to be licensed, having proved itself highly clinically efficacious in patients with the class-3 gating mutation G551D. The trial pipeline seeks to expand indications for this and to explore the potential of Phe508del correctors. Finally, a number of anti-inflammatory and anti-infective strategies are being pursued. The emerging global problem of antibiotic resistance is leading to exciting alternatives such as biofilm disruption and bacteriophage to be explored.

Researchers Use Gene Therapy to Repair Inner-Ear Defects in Mice

ASHA Leader ◽  
2016 ◽  
Vol 21 (2) ◽  
Keyword(s):  
Gene Therapy ◽  
Inner Ear

Cyclooxygenese-2 promoter can mittigate the lethal toxicity in HSV-TK based adenoviral suicide gene therapy of gastrointestinal cancers

2001 ◽  
Vol 120 (5) ◽  
pp. A10-A10
Author(s):  
M YAMAMOTO ◽  
R ALEMANY ◽  
Y ADACHI ◽  
W GRIZZLE ◽  
D CURIEL
Keyword(s):  
Gene Therapy ◽  
Suicide Gene ◽  
Suicide Gene Therapy ◽  
Gastrointestinal Cancers ◽  
Lethal Toxicity

Antiangiogenic gene therapy delays growth of murine and human pancreatic adenocarcinoma in mice

2001 ◽  
Vol 120 (5) ◽  
pp. A349-A349
Author(s):  
J TSENG ◽  
F FARNEBO ◽  
O KISKER ◽  
C BECKER ◽  
C KUO ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pancreatic Adenocarcinoma ◽  
Human Pancreatic Adenocarcinoma

1107: Evaluation of Cystine Transport in Cultured Human Kidney Cells: Primer to Gene Therapy of Cystinuria

2005 ◽  
Vol 173 (4S) ◽  
pp. 300-300
Author(s):  
Sreedhar Sagi ◽  
Lutz Trojan ◽  
Peter Aiken ◽  
Maurice S. Michel ◽  
Thomas Knoll
Keyword(s):  
Gene Therapy ◽  
Human Kidney ◽  
Kidney Cells

790: Interleukin-2 Enhanced the Anti-Tumor Effect of Adenoviral-Mediated HSV-TK Gene Therapy in Murine Bladder Cancer Model

2005 ◽  
Vol 173 (4S) ◽  
pp. 214-214
Author(s):  
Shuji Terao ◽  
Toshiro Shirakawa ◽  
Kazumasa Goda ◽  
Sadao Kamidono ◽  
Akinobu Gotoh
Keyword(s):  
Gene Therapy ◽  
Bladder Cancer ◽  
Interleukin 2 ◽  
Cancer Model ◽  
Bladder Cancer Model

622: Intravesical Interleukin-12 Gene Therapy in an Orthotopic Bladder Cancer Model

2006 ◽  
Vol 175 (4S) ◽  
pp. 200-201
Author(s):  
Minoru Horinaga ◽  
Kelley Harsch ◽  
Ryuichi Fukuyama ◽  
Warren Heston ◽  
William Larchian
Keyword(s):  
Gene Therapy ◽  
Bladder Cancer ◽  
Interleukin 12 ◽  
Cancer Model ◽  
Bladder Cancer Model

1417: Mesenchymal Stem Cells Alone or Modified with ENOS Restore Erectile Function in the Aged Rat: Cell-Based Gene Therapy for Erectile Dysfunction

2004 ◽  
Vol 171 (4S) ◽  
pp. 373-373
Author(s):  
Trinity J. Bivalacqua ◽  
Mustafa F. Usta ◽  
Hunter C. Champion ◽  
Weiwen Deng ◽  
Philip J. Kadowitz ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Mesenchymal Stem Cells ◽  
Erectile Dysfunction ◽  
Erectile Function ◽  
Aged Rat

972: Antisense CK2 Gene Therapy Induces Regression in Human Bladder Cancer Growing in Athymic Nude Mice

2004 ◽  
Vol 171 (4S) ◽  
pp. 257-258
Author(s):  
Joel Slaton ◽  
Daniel Sloper ◽  
Miriam Taylor ◽  
Alan Davis ◽  
Khalil Ahmed
Keyword(s):  
Gene Therapy ◽  
Bladder Cancer ◽  
Nude Mice ◽  
Human Bladder Cancer ◽  
Human Bladder ◽  
Athymic Nude Mice

967: A Targeted Radiotherapy and Tumour Specific Gene Therapy Approach for Bladder Cancer: Transfection of the Noradrenaline Transporter Gene Under the Control of Telomerase Promoters

2004 ◽  
Vol 171 (4S) ◽  
pp. 256-256
Author(s):  
Natasha E. Fullerton ◽  
Marie Boyd ◽  
Robert J. Mairs ◽  
Susan Ross ◽  
Lesley E.D. Wilson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Bladder Cancer ◽  
Specific Gene ◽  
Transporter Gene ◽  
Targeted Radiotherapy ◽  
Therapy Approach ◽  
Noradrenaline Transporter ◽  
Gene Therapy Approach
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