What Is Gene Therapy? How Does It Work?

Gene therapy is the process of replacing defective genes with healthy ones, adding new genes to help the body fight or treat disease, or deactivating problem genes. It holds the promise to transform medicine and create options for patients who are living with difficult, and even incurable, diseases. Learn how this innovative therapy works.
Source: FDA Consumer Health Information Updates - Category: Consumer Health News Source Type: news

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Fight Aging! provides a weekly digest of news and commentary for thousands of subscribers interested in the latest longevity science: progress towards the medical control of aging in order to prevent age-related frailty, suffering, and disease, as well as improvements in the present understanding of what works and what doesn't work when it comes to extending healthy life. Expect to see summaries of recent advances in medical research, news from the scientific community, advocacy and fundraising initiatives to help speed work on the repair and reversal of aging, links to online resources, and much more. This content is...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
The TWiV team reveals the repertoire of anti-viral antibodies in newborn humans, and a complement protein that binds the adenovirus capsid and prevents release of the viral DNA. Hosts: Vincent Racaniello, Rich Condit, Kathy Spindler, and Brianne Barker Subscribe (free): iTunes, Google Podcasts, RSS, email Become a patron of TWiV! Links for this episode Maternal anti-viral antibodies in newborns (Nat Med) Complement protein blocks adenovirus uncoating (Cell Host Micr) Antibodies neutralize virus inside cells (virology blog) Image credit Letters read on TWiV 544 Timestamps by Jolene. T...
Source: This Week in Virology - MP3 Edition - Category: Virology Authors: Source Type: podcasts
Conclusions: G-SCF decreased the aminotransferases activity, cholesterol level, and glucose level in patients with DMD, which may be important for patients with DMD and metabolic syndrome. PMID: 31001554 [PubMed - in process]
Source: Biomed Res - Category: Research Authors: Tags: Biomed Res Int Source Type: research
Publication date: Available online 17 April 2019Source: Colloids and Surfaces B: BiointerfacesAuthor(s): Marc Suñé-Pou, María J. Limeres, Isaac Nofrerias, Anna Nardi-Ricart, Silvia Prieto-Sánchez, Younes El-Yousfi, Pilar Pérez-Lozano, Encarna García-Montoya, Montserrat Miñarro-Carmona, Josep Ramón Ticó, Cristina Hernández-Munain, Carlos Suñé, Josep Mª Suñé-NegreAbstractThe development of new nanoparticle formulations that are capable of high transfection efficiency without toxicity is essential to provide new tools for ge...
Source: Colloids and Surfaces B: Biointerfaces - Category: Biochemistry Source Type: research
PMID: 31002032 [PubMed]
Source: Current Pharmaceutical Biotechnology - Category: Biotechnology Authors: Tags: Curr Pharm Biotechnol Source Type: research
The $1.2 billion deal is the latest designed to support the flood of gene therapies in development
Source: Chemical and Engineering News - Category: Chemistry Authors: Source Type: research
Novartis AG, which this week announced positive interim trial results for its experimental gene therapy for spinal muscular atrophy, on Friday said investigation is underway into whether a second trial death could be related to the treatment.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Genome editing represents a promising strategy to correct COL7A1 gene mutations that cause recessive dystrophic epidermolysis bullosa (RDEB). Previously, we used programmable nucleases that create double-stranded DNA breaks (DSBs) to repair COL7A1 mutations through homology-directed repair (HDR) with an exogenous repair template. Delivery of this template can be cytotoxic and DSBs induce undesired insertions and deletions (indels) that compete with desired HDR. To overcome these limitations, we used base editors (BE), a CRISPR/Cas9-based system that uses naturally occurring or laboratory-evolved deaminating enzymes to dire...
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
Acne and rosacea are both prevalent inflammatory conditions with negative psychosocial impact and unmet clinical needs. Knowledge of distinct gene expression changes and implicated biologic pathways specific to acne versus rosacea is needed to streamline the selection of candidate compounds for rapid testing and development in the appropriate patient populations. We pooled and compared transcriptomes from N=12 acne patients, N=19 rosacea patients, and N=22 respective controls to obtain differentially expressed genes, then performed Ingenuity upstream regulator analysis to identify compounds highly predicted to target aberr...
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
This study aimed to assess the expression of circRNAs in PBMCs from patients with SLE and healthy individuals by RNA sequencing (RNA-seq). Methods: In total, 128 circRNAs were significantly differentially expressed between these two groups, including 39 upregulated and 89 downregulated circRNAs.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
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