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15:37 EDT 24th September 2018 | BioPortfolio

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Showing "Agilis Biotherapeutics Gene Therapy Research Institution Enter into" PubMed Articles 1–25 of 44,000+

Gene therapy for visual loss: Opportunities and concerns.

Many clinical trials using gene therapy have shown significant therapeutic benefits and exceptional safety records. Increasing evidence is verifying the long sought-after promise that gene therapy will genetically 'cure' some severely disabling diseases. In particular, the first gene therapy bioproduct for RPE65-associated Leber's congenital amaurosis, which was approved by the US Food and Drug Administration in 2017, has provided tremendous encouragement to the field of gene therapy. Recent developments in...

Limited success with clostridium collagenase histolyticum following FDA approval for the treatment of Peyronie's disease.

Collagenase clostridium histolyticum (CCH) was approved for the treatment of Peyronie's disease (PD) in December of 2013. Recently, further research has supported its use. The aim of this study was to objectively evaluate the effects of CCH on penile curvature associated with Peyronie's disease at our high volume institution. Since the approval of CCH for PD, 45 men have undergone between 1 and 4 treatment cycles (mean 3.13, standard deviation (SD) = 0.87) at our institution. Primary as well as secondar...

Radiation Gene-expression Signatures in Primary Breast Cancer Cells.

In breast cancer (BC) care, radiation therapy (RT) is an efficient treatment to control localized tumor. Radiobiological research is needed to understand molecular differences that affect radiosensitivity of different tumor subtypes and the response variability. The aim of this study was to analyze gene expression profiling (GEP) in primary BC cells following irradiation with doses of 9 Gy and 23 Gy delivered by intraoperative electron radiation therapy (IOERT) in order to define gene signatures of response...

Targeting corneal inflammation by gene therapy: Emerging strategies for keratitis.

Inflammation is the underlying process of several diseases within the eye, specifically in the cornea. Current treatment options for corneal inflammation or keratitis, and related neovascularization, are restricted by limited efficacy, adverse effects, and short duration of action. Gene therapy has shown great potential for the treatment of diseases affecting the ocular surface, and major efforts are being targeted to inflammatory mediators and neovascularization, in order to develop potential treatments fo...

Data collection of patient outcomes: one institution's experience.

Patient- and provider-reported outcomes are recognized as important in evaluating quality of care, guiding health care policy, comparative effectiveness research, and decision-making in radiation oncology. Combining patient and provider outcome data with a detailed description of disease and therapy is the basis for these analyses. We report on the combination of technical solutions and clinical process changes at our institution that were used in the collection and dissemination of this data. This initiati...

Immune Gene Therapy and the International Conference on Lymphocyte Engineering (ICLE 2018).

Future AAVenues for In Utero Gene Therapy.

Fetal gene therapy using safe and effective viral vectors no longer remains a distant prospect. Recently in Nature Medicine, Massaro et al. (2018) demonstrated that prenatal intracranial injection of a viral vector results in improved neurologic function, raising the intriguing possibility that in utero gene therapy may be approaching clinical applications.

Lessons learned from lung and liver in-vivo gene therapy: implications for the future.

Ex-vivo gene therapy has had significant clinical impact over the last couple of years and in-vivo gene therapy products are being approved for clinical use. Gene therapy and gene editing approaches have huge potential to treat genetic disease and chronic illness. Areas covered: This article provides a review of in-vivo approaches for gene therapy in the lung and liver, exploiting non-viral and viral vectors with varying serotypes and pseudotypes to target specific cells. Antibody responses inhibiting viral...

Nucleic-acid based gene therapy approaches for sepsis.

Despite advances in overall medical care, sepsis and its sequelae continue to be an embarrassing clinical entity with an unacceptably high mortality rate. The central reason for high morbidity and high mortality of sepsis and its sequelae is the lack of an effective treatment. Previous clinical trials have largely failed to identify an effective therapeutic target to improve clinical outcomes in sepsis. Thus, the key goal favoring the outcome of septic patients is to devise innovative and evolutionary thera...

Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy.

Laser Therapy for Pediatric Burn Scars: Focusing on a Combined Treatment Approach.

Treatment with laser therapy has the potential to greatly improve hypertrophic scarring in individuals who have sustained burn injuries. More specifically, recent research has demonstrated the success of using pulsed dye laser therapy to help reduce redness and postburn pruritus and using ablative fractional CO2 laser therapy to improve scar texture and thickness. This study describes our early experience using laser therapy in our pediatric burn program and details our specific treatment approach when usin...

Effective gene therapy for hemophilia, at last….

Recent efforts at gene therapy for haemophilia A and B using AAV-derived vectors show durable expression of coagulation factors at significant levels, resulting in almost complete correction of the phenotype. This is the first success of gene therapy for a major hereditary disorder, and shows how continuous improvement of many components of the system has finally succeeded. Although these results must be confirmed with more patients and longer durations, they constitute a significant accomplishment for this...

Correction to: Recent Progress on Genetic Diagnosis and Therapy for β-Thalassemia in China and Around the World, by Zhang J, Yan J, and Zeng F. Hum Gene Ther 2018;29(2):197-203. DOI: 10.1089/hum.2017.228.

Visual Field Variability after Gene Therapy for Leber's Hereditary Optic Neuropathy.

To assess changes in visual field (VF) values after gene therapy for Leber's hereditary optic neuropathy (LHON).

Gene therapy in optic nerve disease.

Highlight some of the recent advances in gene therapy and gene modification for optic nerve disease to promote axon regeneration, neuroprotection, and increased visual functioning.

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Nice.

Viral vectors are rapidly being developed for a range of applications in research and gene therapy. Prototype foamy virus (PFV) vectors have been described for gene therapy, although their use has mainly been restricted to ex vivo stem cell modification. Here we report direct in vivo transgene delivery with PFV vectors carrying reporter gene constructs. In our investigations, systemic PFV vector delivery to neonatal mice gave transgene expression in the heart, xiphisternum, liver, pancreas, and gut, where...

Gene Therapy Briefs.

British Society for Gene and Cell Therapy Annual Conference Friday 27 April 2018 Regent's Conference Centre, London, UK

Diabetic complication could get a gene therapy boost.

Retinal gene therapy.

Inherited retinal diseases are the leading cause of sight impairment in people of working age in England and Wales, and the second commonest in childhood. Gene therapy offers the potential for benefit.

Synthetic Biology and Engineered Live Biotherapeutics: Toward Increasing System Complexity.

Recent advances in synthetic biology and biological system engineering have allowed the design and construction of engineered live biotherapeutics targeting a range of human clinical applications. In this review, we outline how systems approaches have been used to move from simple constitutive systems, where a single therapeutic molecule is expressed, to systems that incorporate sensing of the in vivo environment, feedback, computation, and biocontainment. We outline examples where each of these capabiliti...

Association between ABCA1 gene polymorphisms and the therapeutic response to donepezil therapy in Han Chinese patients with Alzheimer's disease.

It is reported that ABCA1, which plays a key role in cholesterol transport and apolipoprotein E (APOE) metabolism in the brain, is related to Alzheimer's disease. However, few studies have focused on the relationship between the ABCA1 gene and the therapeutic response to donepezil (DNP), which has been shown to be related to reduced sAPP production.This study evaluated the association between the ABCA1 gene polymorphism and the clinical response to donepezil therapy in Han Chinese patients with Alzheimer's ...

Comparison of the editing patterns and editing efficiencies of TALEN and CRISPR-Cas9 when targeting the human CCR5 gene.

The human C-C chemokine receptor type-5 (CCR5) is the major transmembrane co-receptor that mediates HIV-1 entry into target CD4+ cells. Gene therapy to knock-out the CCR5 gene has shown encouraging results in providing a functional cure for HIV-1 infection. In gene therapy strategies, the initial region of the CCR5 gene is a hotspot for producing functional gene knock-out. Such target gene editing can be done using programmable endonucleases such as transcription activator-like effector nucleases (TALEN) or...

Disease-modifying Osteoarthritis Treatment with Interleukin-1 Receptor Antagonist Gene Therapy in Small and Large Animal Models.

Gene therapy holds great promise for the treatment of osteoarthritis (OA) because a single intraarticular injection can lead to long-term expression of therapeutic proteins within the joint. Here we report the development of a helper-dependent adenovirus (HDAd)-mediated intraarticular gene therapy approach for long-term expression of interleukin-1 receptor antagonist (IL-1Ra) for sustained symptomatic and disease-modifying OA therapy.

Gene Therapy and Virotherapy of Gliomas.

Despite many recent advances in the management of gliomas, such as aggressive surgical resection, chemoradiotherapy, antiangiogenic therapy, and molecular targeted therapy, the survival of patients with high-grade neoplasms remains dismal. Gene therapy and oncolytic virotherapy have emerged as highly promising strategies for treatment of malignant brain tumors due to recent progress in understanding of the underlying cancer biology as well as improved techniques for genetic modification of potential therape...

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