PubMed Journals Articles About "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" - Page: 3 RSS

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Showing "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" PubMed Articles 51–75 of 31,000+

Needle EMG, a jigsaw to disclose lipid storage myopathy due to Multiple Acyl-CoA Dehydrogenase Deficiency, a case report.

Multiple acyl-CoA dehydrogenase deficiency (MADD) is a rare autosomal recessive inborn error of metabolism. The late-onset MADD is frequently caused by mutations in ETFDH gene. Due to its clinical heterogeneity, diagnosis and treatment of late-onset MADD are often delayed. The authors described a previously healthy 40-year-old Thai female presenting with subacute severe weakness of bulbar-limb muscles and elevated serum CK. The authors emphasized the importance of needle EMG and prompt muscle histopathologi...

Predictors of Participation and Autonomy in People With Multiple Sclerosis.

Because multiple sclerosis (MS) affects many life areas, it is important to know how participation and autonomy are associated with the perceived impact of MS on everyday life.

Genetic therapies for hearing loss: Accomplishments and remaining challenges.

More than 15 years have passed since the official completion of the Human Genome Project. Predominantly due to this project, over one hundred genes have now been linked to hearing loss. Although major advancements have been made in the understanding of underlying pathologies in deafness as a consequence of these gene discoveries, biological treatments for these conditions are still not available and current treatments rely on amplification or prosthetics. A promising approach for developing treatments for g...

The promise of miRNA replacement therapy for Hepatocellular carcinoma.

MicroRNA modulation therapy has shown great promise to treat hepatocellular carcinoma (HCC).

geneCo: A visualized comparative genomic method to analyze multiple genome structures.

In comparative and evolutionary genomics, a detailed comparison of common features between organisms is essential to evaluate genetic distance. However, identifying differences in matched and mismatched genes among multiple genomes is difficult using current comparative genomic approaches due to complicated methodologies or the generation of meager information from obtained results. This study describes a visualized software tool, geneCo (gene Comparison), for comparing genome structure and gene arrangement...

A safe and effective treatment: surgery combined with photodynamic therapy for multiple basal cell carcinomas.

SOX4: The Unappreciated Oncogene.

SOX4 is an essential developmental transcription factor that regulates stemness, differentiation, progenitor development, and multiple developmental pathways including PI3K, Wnt, and TGFβ signaling. The SOX4 gene is frequently amplified and overexpressed in over 20 types of malignancies, and multiple lines of evidence support that notion that SOX4 is an oncogene. Its overexpression is due to both gene amplification and to activation of PI3K, Wnt, and TGFβ pathways that SOX4 regulates. SOX4 interacts with ...

Leveraging Single-Cell RNA Sequencing Experiments to Model Intratumor Heterogeneity.

Many cancers can be treated with targeted therapy. Almost inevitably, tumors develop resistance to targeted therapy, either from pre-existence or by evolving new genotypes and traits. Intratumor heterogeneity serves as a reservoir for resistance, which often occurs as a result of the selection of minor cellular subclones. On the level of gene expression, clonal heterogeneity can only be revealed using high-dimensional single-cell methods. We propose using a general diversity index (GDI) to quantify heteroge...

Single- Versus Multiple-Fraction Radiation Therapy for Painful Bone Metastases: A Systematic Review and Meta-analysis of Nonrandomized Studies.

Single-fraction radiation therapy (RT) is a convenient and cost-effective regimen for the palliation of painful bone metastases, but is still underused. Randomized controlled trials comparing single- versus multiple-fraction RT are limited by generalizability. We compared the pain response rates after single- versus multiple-fraction RT in nonrandomized studies.

Lipid nanoparticles for delivery of therapeutic RNA oligonucleotides.

Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNP). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and s...

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...

Therapeutic potential of CRISPR/Cas9 gene editing in engineered T-cell therapy.

Cancer patients have been treated with various types of therapies, including conventional strategies like chemo-, radio-, and targeted therapy, as well as immunotherapy like checkpoint inhibitors, vaccine and cell therapy etc. Among the therapeutic alternatives, T-cell therapy like CAR-T (Chimeric Antigen Receptor Engineered T cell) and TCR-T (T Cell Receptor Engineered T cell), has emerged as the most promising therapeutics due to its impressive clinical efficacy. However, there are many challenges and obs...

A randomized pragmatic trial of telephone-delivered cognitive behavioral-therapy, modafinil, and combination therapy of both for fatigue in multiple sclerosis: The design of the "COMBO-MS" trial.

Fatigue is one of the most common and disabling chronic symptoms in multiple sclerosis (MS). Optimization of available treatments for MS-related fatigue has been stymied by lack of comparative effectiveness research that focuses on real-world treatment delivery methods and potential modification of treatment effect by other chronic MS symptoms or disability level. This report describes the design of a patient centered, comparative effectiveness trial of cognitive behavioral-therapy (CBT), modafinil, and com...

Value-based Payment Reform in a Managed Care Environment: Innovator States' Experiences with Episodes of Care.

Non-viral nucleic acid delivery to central nervous system and brain tumor.

Gene therapy is a rapidly emerging remedial route for many serious incurable diseases, such as central nervous system (CNS) diseases. Currently, nucleic acid medicines including DNAs encoding therapeutic or destructive proteins, siRNAs or miRNAs, have been successfully delivered to the CNS with gene delivery vectors via various routes of administration and subsequently exhibited remarkable therapeutic efficiency. Among these vectors, non-viral vectors are favorable for delivering genes into CNS due to many ...

Antibacterial effect of S-Porphin sodium photodynamic therapy on Staphylococcus aureus and multiple drug resistance Staphylococcus aureus.

Antibacterial photodynamic therapy (aPDT) has been proposed as an alternative strategy to inactivate bacteria. This study was designed to investigate the antibacterial effect of a novel photosensitizer S-Porphin sodium (S-PS) on plankton and biofilm cultures of Staphylococcus aureus (S. aureus) and its multiple drug resistance strain S. aureus (MDR S. aureus).

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...

Gene therapy and cell therapy for the management of radiation damages to healthy tissues: Rationale and early results.

Nowadays, ionizing radiations have numerous applications, especially in medicine for diagnosis and therapy. Pharmacological radioprotection aims at increasing detoxification of free radicals. Radiomitigation aims at improving survival and proliferation of damaged cells. Both strategies are essential research area, as non-contained radiation can lead to harmful effects. Some advances allowing the comprehension of normal tissue injury mechanisms, and the discovery of related predictive biomarkers, have led to...

A Genetics Learning Program for Nurses Caring for Children Treated With Ex Vivo Autologous Gene Therapy.

As our growing knowledge of genetics and genomics continues to inform, change, and customize health care, an understanding of genetics and genomics is now central to up-to-date and proficient nursing practice. There is a growing need for relevant nursing educational programs that aid practicing nurses in securing genetics/genomics knowledge and an understanding of gene therapy. This article describes a day-long, evidence-based, hands-on genetics learning program developed specifically for practicing nurses ...

IM-UNITI at Three Years: Stellar Stelara® or Stardust? The Efficacy, Safety, and Immunogenicity of Ustekinumab Treatment of Crohn's Disease.

Development, Reliability, and Validity of the Multiple Errands Test Home Version (MET-Home) in Adults With Stroke.

Our objective was to perform initial psychometric analysis of the Multiple Errands Test Home Version (MET-Home), which was designed to assess the influence of poststroke executive dysfunction on in-home task performance.

Dry AMD - Cellular and Genetic Therapies.

The growing incidence of neurodegenerative diseases is based on our increasingly aging society as well as the difficulties in establishing defined therapy regimens. For dry age-related macular degeneration (AMD) and the later stage of geographic atrophy (GA), various treatment options exist that only decelerate the progression of the disease. However, no therapy is currently available that can restore the degenerated retinal pigment epithelium (RPE) and/or photoreceptor cells. Cellular and gene-based approa...

A unique bZIP transcription factor imparting multiple stress tolerance in Rice.

Rice productivity is adversely affected by environmental stresses. Transcription factors (TFs), as the regulators of gene expression, are the key players contributing to stress tolerance and crop yield. Histone gene binding protein-1b (OsHBP1b) is a TF localized within the Saltol QTL in rice. Recently, we have reported the characterization of OsHBP1b in relation to salinity and drought tolerance in a model system tobacco. In the present study, we over-express the full-length gene encoding OsHBP1b in the hom...

Fecal and Blood Microbial 16s rRNA Gene Alterations in Chinese Patients with Multiple System Atrophy and Its Subtypes.

To explore the alterations of microbial 16s ribosomal (rRNA) gene in the feces and blood of Chinese patients with multiple system atrophy (MSA) and its relationships with clinical features.

Response to First Cycle Is the Major Predictor of Long-Term Response to Lenalidomide and Dexamethasone Therapy in Relapsed and Refractory Multiple Myeloma: Can We Spare Patients the Toxicity and Costs of Additional Agents?

Lenalidomide plus dexamethasone (Ld) is still considered an option of care for some selected patients with relapsed or refractory multiple myeloma (RRMM), despite the proven superiority of lenalidomide-based triplet therapy. Up to 20% of patients obtain long-term benefit from Ld alone. The aim of this multicenter retrospective study was to identify and characterize those with good response to Ld salvage therapy, defined as progression-free survival lasting more than 24 months.

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