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PubMed Journals Articles About "Gene Therapy Clinical Study In Adult PKU" RSS

04:44 EDT 16th June 2019 | BioPortfolio

Gene Therapy Clinical Study In Adult PKU PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Gene Therapy Clinical Study In Adult PKU articles that have been published worldwide.

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Showing "Gene Therapy Clinical Study Adult" PubMed Articles 1–25 of 94,000+

The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world.

In this first in a series of round table meetings, the 1st World Federation of Hemophilia Gene Therapy Round Table was convened to initiate a global dialogue on the expected challenges and opportunities that a disruptive therapy, such as gene therapy, will bring to the haemophilia community. Perspectives from key stakeholder groups, including healthcare professionals, regulators, payors, people with hemophilia and pharmaceutical industry representatives, were sought in the identification of the key issues w...


Adeno-associated virus vector as a platform for gene therapy delivery.

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growth of the gene therapy field. Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two ...

Gene therapy for blood diseases.

Genetic diseases affecting proteins and cells composing the blood may be treated by gene therapy using gene addition or gene editing methods. Protein deficiencies (e.g. hemophilia) are being approached using in vivo gene delivery by adeno-associated virus (AAV) vectors for therapeutic gene addition or gene editing. Blood cell diseases (e.g. sickle cell disease) are being approached using ex vivo gene addition or gene editing to treat isolated blood-forming hematopoietic stem cells or T cells that are then r...


Polycations for Gene Delivery: Dilemmas and Solutions.

Gene therapy has been a promising strategy for treating numerous gene-associated human diseases by altering specific gene expressions in pathological cells. Application of non-viral gene delivery is hindered by various dilemmas encountered in systemic gene therapy. Therefore, solutions must be established to address the unique requirements of gene-based treatment of diseases. This review will particularly highlight the dilemmas in polycations-based gene therapy by systemic treatment. Several promising strat...

Gene Therapy Briefs.

Repairing the Brain: Gene Therapy.

In vivo gene therapy for neurodegenerative disorders has turned out to be a formidable challenge. It is a field not much older than twenty years, but we were many who would have predicted a much easier path towards the clinic using this treatment modality. For Parkinson's disease patients, this has meant a frustrating wait, seeing many promising therapies being forgotten after a few pre-clinical proof-of-concept studies. The reasons for this are both scientific and economical. However, this is slowly but su...

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.

Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly considered for rare inherited disorders, gene therapy may open treatment opportunities for more challenging and complex diseases such as Alzheimer's or Parkinson's disease. Today, examples of striking clinical proof of concept, the first gene therapy drugs coming onto the market, and the emergence of powerful new molecular tools have broadened the number of avenues to target neurological disorders but have also highlight...

Long-term outcomes of cardiac resynchronization therapy in adult congenital heart disease.

Randomized, controlled trials of cardiac resynchronization therapy (CRT) excluded patients with adult congenital heart disease (ACHD). We sought to explore long-term clinical outcomes.

Gene Therapy: Paving New Roads in the Treatment of Hemophilia.

Hemophilia is a monogenic disease with robust clinicolaboratory correlations of severity. These attributes coupled with the availability of experimental animal models have made it an attractive model for gene therapy. The road from animal models to human clinical studies has heralded significant successes, but major issues concerning a previous immunity against adeno-associated virus and transgene optimization remain to be fully resolved. Despite significant advances in gene therapy application, many questi...

Feasibility and utility of a panel testing for 114 cancer-associated genes in a clinical setting: A hospital-based study.

Next generation sequencing (NGS) of tumor tissue (i.e., clinical sequencing) can guide clinical management by providing information about actionable gene aberrations that have diagnostic and therapeutic significance. Here, we performed a hospital-based prospective study (TOP-GEAR project, 2 stage) to investigate the feasibility and utility of NGS-based analysis of 114 cancer-associated genes (the NCC Oncopanel test). We examined 230 cases (comprising > 30 tumor types) of advanced solid tumors, all of which ...

Advances in the techniques and methodologies of cancer gene therapy.

Cancer is the second leading cause of mortality worldwide after cardiovascular diseases, predominantly due to the lack of early symptoms and early diagnosis, and high relapse rate after radical surgery and conventional therapies. Therefore, novel approaches such as gene therapy have raised hope to significantly improve the survival rate of patients with cancers. This review aims to provide up-to-date information concerning gene therapy including improved vectors, suicide genes, cancer suppressor genes, anti...

Developing a novel cholesterol-based nanocarrier with high transfection efficiency and serum compatibility for gene therapy.

Primary cells are sensitive to culture conditions, which can be more difficult to get efficient transfection. The purpose of this study is to develop a serum-compatible cholesterol-based nanocarrier for delivering therapeutic nucleic acids into cells efficiently for future clinical gene therapy.

Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives.

Ocular gene therapy has entered into clinical practice. Although viral vectors are currently the best option to replace and/or correct genes, the optimal method to deliver these treatments to the retinal pigment epithelial (RPE) cells and/or photoreceptor cells remains to be improved to increase transduction efficacy and reduce iatrogenic risks. Beyond viral-mediated gene replacement therapies, nonviral gene delivery approaches offer the promise of sustained fine-tuned expression of secreted therapeutic pro...

Biomarkers and clinical rating scales for sodium pyruvate therapy in patients with mitochondrial disease.

Biomarkers and two clinical rating scales-the Japanese mitochondrial disease-rating scale (JMDRS) and Newcastle mitochondrial disease adult scale (NMDAS)-are clinically used when treating patients with mitochondrial disease. We explored the biomarker(s) and clinical rating scale(s) that are appropriate in preparing the protocol for a future clinical trial of sodium pyruvate (SP) therapy. A 48-week, prospective, single-centre, exploratory, clinical study enrolled 11 Japanese adult patients with genetically, ...

Transparenchymal renal pelvis injection of recombinant AAV9 vectors is a practical approach for gene delivery in the kidney.

Gene therapy has great potential in treating human diseases, but little progress has been made in preclinical and clinical studies of renal diseases. To find an effective gene delivery approach in the kidney, we developed transparenchymal renal pelvis injection. Using AAV9 vectors, we evaluated the gene delivery efficiency and safety of this administration method. The results showed that the exogenous gene was expressed in the tubular epithelial cells of the injected kidney, with a much lower expression lev...

CIK cell-based delivery of recombinant adenovirus KGHV500 carrying the anti-p21Ras scFv gene enhances the anti-tumor effect and safety in lung cancer.

Adenovirus (Ads) is one of the most popular vectors used in gene therapy for the treatment of cancer. However, systemic therapy is limited by circulating antiviral antibodies and poor viral delivery in vivo. In this study, we used cytokine-induced killer (CIK) cells as delivery vehicles of Ads KGHV500 carrying the anti-p21Ras scFv gene to treat Ras gene-related lung cancer and investigate the anti-tumor effect in vitro and in vivo.

Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments.

Although high upfront costs for the high value of gene therapy have resulted in concerns about sufficient reimbursement to allow patient access to these therapies, the significant benefits of gene therapies will not be realized unless patients have access to them. Stakeholders are discussing these issues, and the payment models being developed for the newly approved gene therapies provide an early indication of the flexibility that will be needed from treatment manufacturers, payers, and policy makers to op...

AntiHIV/AIDS drug 6HP: antiviral activity, pre-clinical study. Efficiency in adult HIV-infected patients.

The new domestic antiretroviral drug 6HP, which is ammonium-3'-azido-3'-deoxythymidine-5'-carbomoylphosphonate, shows a high level of anti-HIV activity in cultures of lymphoblastoid cells. In a organism, the 6HP is converted to azidothymidine, and the its pharmacokinetic parameters indicate a prolonged nature of action of this compound in vivo. It is an important indicator that allows to formulate optimal therapeutic regimens during clinical application of 6HP. The complex of its antiviral properties and th...

Larotrectinib in adult patients with solid tumours: a multicentre, open-label, phase I dose-escalation study.

NTRK1, NTRK2 and NTRK3 gene fusions (NTRK gene fusions) occur in a range of adult cancers. Larotrectinib is a potent and highly selective ATP-competitive inhibitor of TRK kinases and has demonstrated activity in patients with tumours harbouring NTRK gene fusions.

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.

Wiskott-Aldrich syndrome is a rare, life-threatening, X-linked primary immunodeficiency characterised by microthrombocytopenia, infections, eczema, autoimmunity, and malignant disease. Lentiviral vector-mediated haemopoietic stem/progenitor cell (HSPC) gene therapy is a potentially curative treatment that represents an alternative to allogeneic HSPC transplantation. Here, we report safety and efficacy data from an interim analysis of patients with severe Wiskott-Aldrich syndrome who received lentiviral vect...

Randomized Phase II Trial Comparing Site-Specific Treatment Based on Gene Expression Profiling With Carboplatin and Paclitaxel for Patients With Cancer of Unknown Primary Site.

Although gene expression profiling is a promising diagnostic technique to determine the tissue of origin for patients with cancer of unknown primary site (CUP), no clinical trial has evaluated yet site-specific therapy directed by this approach compared with empirical chemotherapy. We therefore performed a randomized study to assess whether such site-specific therapy improves outcome compared with empirical chemotherapy in previously untreated patients with CUP.

Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer.

Advances in DNA- and RNA-based technologies have made gene therapy suitable for many lung diseases, especially those that are hereditary. The main objective of gene therapy is to deliver an adequate amount of gene construct to the intended target cell, achieve stable transduction in target cells, and to produce a clinically therapeutic effect. This review focuses on the cellular organization in the normal lung and how gene therapy targets the specific cell types that are affected by pulmonary disorders caus...

A Randomized, Double-Blind Trial Comparing the Pharmacokinetics of CT-P16, a Candidate Bevacizumab Biosimilar, with its Reference Product in Healthy Adult Males.

CT-P16 is a candidate biosimilar of bevacizumab, a monoclonal antibody targeting vascular endothelial growth factor that is used in the treatment of a range of advanced solid cancers.

Detection of tumor NTRK gene fusions to identify patients who may benefit from TRK inhibitor therapy.

Chromosomal rearrangements involving the NTRK1, NTRK2, and NTRK3 genes (NTRK gene fusions), which encode the TRKA, TRKB, and TRKC receptor tyrosine kinases, act as oncogenic drivers in a broad range of pediatric and adult tumor types. NTRK gene fusions have been shown to be actionable genomic events that are predictive of response to TRK kinase inhibitors, making their routine detection an evolving clinical priority. In certain exceedingly rare tumor types, NTRK gene fusions may be seen in the overwhelming ...

Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9.

Hemophilia B (HB) is a life-threatening inherited disease caused by mutations in the FIX gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic nature, HB is one of the primary targets for gene therapy. Indeed, successful correction of HB has been shown in clinical trials using gene therapy approaches. However, application of these strategies to non-adult patients is limited due to high cell turnover as young patients develop, resulting in vector dilution and subsequent ...


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