Ocular gene therapy trials due to report this year; Keeping an eye on clinical trials in 2008

Bainbridge, James; Ali, Robin R.

doi:10.1038/gt.2008.28

Cited by 10 publications

(3 citation statements)

References 5 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Can this be cured? Outstanding work, by Ali, Bainbridge, Moore, and colleagues at Moorfields Eye Hospital and in the Division of Inherited Eye Disease, UCL, is making real progress here (102). This is possibly the most clinically advanced topic covered in this review.…”

Section: Canine Modelsmentioning

confidence: 93%

Animal Models for Target Diseases in Gene Therapy — using DNA and siRNA Delivery Strategies

Blagbrough

Zara

2008

Pharm Res

View full text Add to dashboard Cite

Nanoparticles, including lipopolyamines leading to lipoplexes, liposomes, and polyplexes are targeted drug carrier systems in the current search for a successful delivery system for polynucleic acids. This review is focused on the impact of gene and siRNA delivery for studies of efficacy, pharmacodynamics, and pharmacokinetics within the setting of the wide variety of in vivo animal models now used. This critical appraisal of the recent literature sets out the different models that are currently being investigated to bridge from studies in cell lines through towards clinical reality. Whilst many scientists will be familiar with rodent (murine, fecine, cricetine, and musteline) models, few probably think of fish as a clinically relevant animal model, but zebrafish, madake, and rainbow trout are all being used. Larger animal models include rabbit, cat, dog, and cow. Pig is used both for the prevention of foot-and-mouth disease and human diseases, sheep is a model for corneal transplantation, and the horse naturally develops arthritis. Non-human primate models (macaque, common marmoset, owl monkey) are used for preclinical gene vector safety and efficacy trials to bridge the gap prior to clinical studies. We aim for the safe development of clinically effective delivery systems for DNA and RNAi technologies.

show abstract

Section: Canine Modelsmentioning

confidence: 93%

Animal Models for Target Diseases in Gene Therapy — using DNA and siRNA Delivery Strategies

Blagbrough

Zara

2008

Pharm Res

View full text Add to dashboard Cite

show abstract

“…Selective delivery can be made possible by the inclusion of antibody on the vector surface, which specifically binds a surface antigen uniquely associated with the target cell; or vector engineered with a specific hormone that can only bind to cells displaying the hormone receptor [2]. The therapeutic potential of gene therapy includes curing in-born errors of metabolism, or conditions induced by the presence of a defective copy of a specific gene (s), including cancer, AIDS, cystic fibrosis, haemophilia, neurological disorder, or retinal degeneration [2,84-86]. …”

Section: Scientific and Technological Innovations In Biopharmaceuticalsmentioning

confidence: 99%

Cell Engineering and Molecular Pharming for Biopharmaceuticals

Abdullah¹,

Rahmah²,

Sinskey³

et al. 2008

Open Med Chem J

View full text Add to dashboard Cite

Biopharmaceuticals are often produced by recombinant E. coli or mammalian cell lines. This is usually achieved by the introduction of a gene or cDNA coding for the protein of interest into a well-characterized strain of producer cells. Naturally, each recombinant production system has its own unique advantages and disadvantages. This paper examines the current practices, developments, and future trends in the production of biopharmaceuticals. Platform technologies for rapid screening and analyses of biosystems are reviewed. Strategies to improve productivity via metabolic and integrated engineering are also highlighted.

show abstract

“…Recombinant adeno-associated virus-based delivery of RPE65 cDNA to dogs by subretinal injection restored retinal function and provided stable long-term gene expression for more than 3 years. This result was further translated into clinical trials in humans [4–6]. However, viral vectors generally suffer from safety issues such as muta-genesis and toxicity [7].…”

mentioning

confidence: 99%

Polymeric Vectors for Ocular Gene Delivery

Tamboli

Mishra

Mitra³

2011

Therapeutic Delivery

View full text Add to dashboard Cite

Gene therapy holds promise for the treatment of many inherited and acquired diseases of the eye. Successful ocular to targeted cells with minimal toxicity. A major gene therapy interventions depend on challenge is to overcome both intracellular and extracellular barriers associated with ocular gene delivery. Numerous viral and nonviral vectors were explored to improve transfection efficiency. Among nonviral delivery systems, polymeric vectors have gained significant attention in recent years owing to their nontoxic and non-immunogenic nature. Polyplexes or nanoparticles can be prepared by interaction of cationic polymers with DNA, which facilitate cellular uptake, endolysosomal escape and nuclear entry through active mechanisms. Chemical modification of these polymers allows for the generation of flexible delivery vectors with desirable properties. In this article several synthetic and natural polymeric systems utilized for ocular gene delivery are discussed.

show abstract

Ocular gene therapy trials due to report this year; Keeping an eye on clinical trials in 2008

Cited by 10 publications

References 5 publications

Animal Models for Target Diseases in Gene Therapy — using DNA and siRNA Delivery Strategies

Animal Models for Target Diseases in Gene Therapy — using DNA and siRNA Delivery Strategies

Cell Engineering and Molecular Pharming for Biopharmaceuticals

Polymeric Vectors for Ocular Gene Delivery

Contact Info

Product

Resources

About