PubMed Journals Articles About "JPM19 Spark Looks Scale Manufacturing Gene Therapy Matures" RSS

01:24 EDT 21st March 2019 | BioPortfolio

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Showing "JPM19 Spark looks scale manufacturing gene therapy matures" PubMed Articles 1–25 of 29,000+

Shared Platform for Antibiotic Research and Knowledge: A Collaborative Tool to SPARK Antibiotic Discovery.

The discovery of urgently needed antibiotics is hindered by challenges to information sharing. To help address this challenge, The Pew Charitable Trusts launched SPARK: the Shared Platform for Antibiotic Research and Knowledge. SPARK is an online, publicly available, interactive database designed to help scientists build on previous research and generate new insights to advance the field's understanding of Gram-negative permeability. This Viewpoint details how data are selected and integrated into the plat...

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...

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...

SeQuiLa: An elastic, fast and scalable SQL-oriented solution for processing and querying genomic intervals.

Efficient processing of large-scale genomic datasets has recently become possible due to the application of 'big data' technologies in bioinformatics pipelines.We present SeQuiLa - a distributed, ANSI SQL compliant solution for speedy querying and processing of genomic intervals that is available as an Apache Spark package. Proposed range join strategy is significantly (~22x) faster than the default Apache Spark implementation and outperforms other state-of-the-art tools for genomic intervals processing.

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...

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...

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...

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...

Unleashing the Therapeutic Potential of CAR-T Cell Therapy Using Gene-Editing Technologies.

Chimeric antigen receptor (CAR) T-cell therapy, an emerging immunotherapy, has demonstrated promising clinical results in hematological malignancies including B-cell malignancies. However, accessibility to this transformative medicine is highly limited due to the complex process of manufacturing, limited options for target antigens, and insufficient anti-tumor responses against solid tumors. Advances in gene-editing technologies, such as the development of Zinc Finger Nucleases (ZFNs), Transcription Activat...

An important step on the long path to clinical application of in utero gene therapy.

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.


Choroideremia (CHM) is a rare inherited retinal degeneration resulting from mutation of the CHM gene, which results in absence of functional Rab escort protein 1 (REP1). We evaluated retinal gene therapy with an adeno-associated virus vector that used to deliver a functional version of the CHM gene (AAV2-REP1).

Gene Therapy Briefs.

Gene Therapy Briefs.

Effects of CYP2C19*17 polymorphisms on the efficacy and safety of bromodigyrochlorophenylbenzodiazepine in patients with anxiety disorder and comorbid alcohol use disorder.

Background Bromodihydrochlorophenylbenzodiazepine (Phenazepam®) is used in the therapy of anxiety disorders in patients with alcohol dependence. However, Phenazepam therapy often turns out to be ineffective, and some patients develop dose-related adverse drug reactions (ADR): severe sedation, dizziness, headache, dyspepsia, falling, etc. That ensures the effectiveness of this category of patients. Despite the popularity of Phenazepam® as an anxiolytic drug, there is currently no accurate data on its biotr...

Diabetic complication could get a gene therapy boost.

Redefining influenza seasonality at a global scale and aligning it to the influenza vaccine manufacturing cycle: a descriptive time series analysis.

Annual seasonal influenza vaccine manufacturing cycles align with temperate country seasonality in each hemisphere, yet influenza seasonality is poorly defined for many countries. The study introduces a novel and universal approach to defining and classifying seasonality that can be used to classify any country's influenza vaccine cycle alignment.

Ensuring Product Quality, Consistency and Patient Supply over Time for a Large-Volume Biologic: Experience with Remicade.

Biologics are produced from living organisms in complex, multi-stage manufacturing processes and contain inherent variability, which must be understood and controlled during manufacturing to avoid unexpected changes in key quality attributes that may contribute to clinically meaningful differences. The process must also meet large commercial demand, while simultaneously being able to accommodate change without sacrificing product consistency. The four key components of successful biologics manufacturing are...

Ten-Year Evaluation of the TOYOTA Prehospital Stroke Scale for Tissue Plasminogen Activator Intravenous Therapy in the Real World.

Emergency medical services are an important part of acute stroke management. We devised a prehospital stroke scale, the TOYOTA prehospital stroke scale for tissue plasminogen activator (t-PA) intravenous therapy (TOPSPIN) for triaging patients with ischemic stroke and especial indications for intravenous t-PA therapy in December 2006; this scale comprises 5 items including consciousness, atrial fibrillation, language disorder, disturbance of the upper extremities, and disturbance of the lower extremities. T...

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...

Branch-PCR constructed TP53 gene nanovector for potential cancer therapy.

A novel and efficient branch PCR strategy has been used to construct a TP53 gene nanovector based on a pair of trimers as primers, which showed unique advantages compared to other existing systems for gene delivery and effective potential cancer therapy.

Progress in gene therapy using oncolytic vaccinia virus as vectors.

Vaccinia virus was widely used in the World Health Organization's smallpox eradication campaign and is currently a promising vector for gene therapy owing to its unique characteristics. Vaccinia virus can selectively replicate and propagate productively in tumor cells, resulting in oncolysis. In addition, rapid viral particle production, wide host range, large genome size (approximately 200 kb), and safe handling render vaccinia virus a suitable vector for gene therapy.

The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases.

Review barriers to effective transduction of cells in the subretinal plane during gene therapy surgery for inherited retinal dystrophies (IRD).

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...

Single-Cell Allele-Specific Gene Expression Analysis.

Allele-specific expression is traditionally studied by bulk RNA sequencing, which measures average gene expression across cells. Single-cell RNA sequencing (scRNA-seq) allows the comparison of expression distribution between the two alleles of a diploid organism, and characterization of allele-specific bursting. Here we describe SCALE, a bioinformatic and statistical framework for allele-specific gene expression analysis by scRNA-seq. SCALE estimates genome-wide bursting kinetics at the allelic level while...

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