Perspective in Progress of Cardiovascular Gene Therapy by Ryuichi Morishita

“…This review focuses on the future potential of gene therapy for the treatment of cardiovascular disease. …”
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Progress in gene therapy for prostate cancer by Kamran Ali Ahmed, Brian James Davis, Torrence M Wilson, Gregory A Wiseman, Mark J Federspiel, John C Morris

Subjects: “…Gene Therapy…”
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Viral Vector-Based Gene Therapy by Xuedan Li, Yang Le, Zhegang Zhang, Xuanxuan Nian, Bo Liu, Xiaoming Yang

Subjects: “…gene therapy…”
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Bringing gene therapy to regenerative medicine by Elizabeth R. Balmayor

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Gene therapy for cleft lip and palate by Mohammad Khursheed Alam, Anas Imran Arshad

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The development of gene therapy for diseases of the lung. by Gill, DR, Davies, L, Pringle, I, Hyde, S

“…The development of a successful gene therapy has many stages, including preclinical testing in animal models and proof of principle clinical studies. …”

Gene therapy targeting inflammation in atherosclerosis. by Van-Assche, T, Huygelen, V, Crabtree, M, Antoniades, C

“…Therefore, although premature to discuss about effective "gene therapy" in atherosclerosis at a clinical level, gene delivery techniques opened new horizons in cardiovascular research, and the development of new vectors may allow their extensive use in clinical trials in the future.…”

Neuroprotection gene therapy in retinal degeneration by Shan, H

“…The neuroprotection of XIAP has been demonstrated in various neurodegenerative models. Retinal gene therapy based on adeno-associated virus (AAV) has recently been proven safe and effective in clinical trials of Leber's congenital amaurosis. …”

Gene therapy for pulmonary alveolar proteinosis by Atsumi, N, Pilou, A, Pringle, I, Ashworth, R, Meng, C, Chan, M, Gill, D, Hyde, S, Morgan, C, Alton, E, Griesenbach, U

Gene therapy meets vaccine development. by Collins, M, Cerundolo, V

“…Our increasing understanding of DC biology, coupled with the growing sophistication of viral vectors developed for gene therapy, makes more rational vaccine design an exciting possibility. …”

Gene therapy targeting to tumor endothelium. by Bazan-Peregrino, M, Seymour, L, Harris, A

“…Tumor vasculature also represents an important site of therapeutic action by the secreted products of antiangiogenic gene therapies that are expressed in non-endothelial cells. …”

Optogenetic gene therapy for vision restoration by Hickey, D

Subjects: “…Gene therapy…”

Cancer gene therapy: The challenge of delivery by Seymour, L

“…Everybody understands gene therapy. It's where healthy genes are used to supplement the loss-of-function of mutated genes, providing simple cures for complex genetic diseases. …”

Surrogate gene therapy for muscular dystrophy by Davies, KE, Chamberlain, JS

“…An engineered truncated gene derived from the dystrophin-related protein (utrophin), prevents pathology without an immune response in an animal model of Duchenne muscular dystrophy (DMD) gene therapy.…”

Immunological responses to retinal gene therapy by Chandler, L

Subjects:

Emerging gene therapies for cystic fibrosis by Miah, K, Hyde, S, Gill, D

“…Overall, gene delivery technologies look more convincing in producing an effective CF gene therapy.</p>…”

Parfitian priority, gene therapy and disability by Wilkinson, D

“…<p>Gene therapies for severe genetic disease are often highly expensive. …”

Clinical applications of retinal gene therapy. by Lipinski, D, Thake, M, Maclaren, R

“…These recent steps have been most significant in bringing gene therapy into the mainstream of ophthalmology. Nevertheless translating retinal gene therapy from animal research into clinical trials is still a lengthy process, including complexities in human retinal diseases that have been difficult to model in the laboratory. …”

Prospects of immune gene therapy for cancer by Hardwick, N, Chan, L, Darling, D, Gaken, J, Galea-Lauri, J

Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective by Michael W. Bolt, Laurence O. Whiteley, Jessica L. Lynch, Brian Lauritzen, Antonio R. Fernández de Henestrosa, Timothy MacLachlan, Peter Ulrich, Binu K. Philip, Prathap Kumar Mahalingaiah, Claudette L. Fuller, David R. Compton

“…Nonclinical development strategies for gene therapies are unique from other modalities. The European Federation of Pharmaceutical Industries and Associates (EFPIA) Gene Therapy Working Group surveyed EFPIA member and nonmember pharmaceutical and biotechnology companies about their current practices for designing and implementing nonclinical toxicology studies to support the development of viral vector-delivered in vivo gene therapies. …”
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