Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus Statement.
Sign Up to like & getrecommendations! Published in 2024 at "JAMA network open"
DOI: 10.1001/jamanetworkopen.2024.29443
Abstract: Importance The introduction of gene therapies into the clinical care landscape for individuals living with sickle cell disease (SCD) represents a momentous achievement with the potential to rewrite the story of the world's most prevalent… read more here.
Keywords: therapy; patient readiness; gene therapy; gene ... See more keywords
An Imperative for Public Sharing of Adverse Events of Gene Therapy Trials.
Sign Up to like & getrecommendations! Published in 2025 at "JAMA neurology"
DOI: 10.1001/jamaneurol.2024.4671
Abstract: This Viewpoint describes the need for public sharing of adverse events associated with genetic therapies to improve patient safety. read more here.
Keywords: adverse events; events gene; public sharing; imperative public ... See more keywords
Utility of In Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity Before Gene Therapy.
Sign Up to like & getrecommendations! Published in 2019 at "JAMA ophthalmology"
DOI: 10.1001/jamaophthalmol.2019.3914
Abstract: Importance Next-generation sequencing can detect variants of uncertain significance (VUSs), for some of which gene therapy would not be advantageous. Therefore, the pathogenicity of compound heterozygous or homozygous variants should be confirmed before bilateral vitrectomy… read more here.
Keywords: vitro mutagenesis; gene therapy; rpe65; pathogenicity ... See more keywords
A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease: The Medicaid Perspective.
Sign Up to like & getrecommendations! Published in 2021 at "JAMA pediatrics"
DOI: 10.1001/jamapediatrics.2020.7140
Abstract: Importance Hundreds of gene therapies are undergoing clinical testing and are likely to be priced more than $1 million per course of treatment. The association that high prices will have with insurance coverage of gene… read more here.
Keywords: budget impact; gene; gene therapy; disease ... See more keywords
Evolution of Gene Therapy for Inborn Errors of Immunity.
Sign Up to like & getrecommendations! Published in 2024 at "JAMA pediatrics"
DOI: 10.1001/jamapediatrics.2024.1116
Abstract: This Viewpoint reviews the history and current state of gene therapy for inborn errors of immunity. read more here.
Keywords: inborn errors; therapy inborn; errors immunity; gene therapy ... See more keywords
Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Sign Up to like & getrecommendations! Published in 2023 at "Annals of clinical and translational neurology"
DOI: 10.1002/acn3.51772
Abstract: OBJECTIVE Mucopolysaccharidosis type IIIA (MPSIIIA) caused by recessive SGSH variants results in sulfamidase deficiency, leading to neurocognitive decline and death. No disease-modifying therapy is available. The AAVance gene therapy trial investigates AAVrh.10 overexpressing human sulfamidase… read more here.
Keywords: intracerebral gene; mucopolysaccharidosis; focal lesions; therapy ... See more keywords
Gene Therapy Based on Nucleic Acid Nanostructure.
Sign Up to like & getrecommendations! Published in 2020 at "Advanced healthcare materials"
DOI: 10.1002/adhm.202001046
Abstract: During the past decades, nucleic acids have been employed for the construction of versatile nanostructures with well-defined shapes and sizes. Owing to the remarkable programmability, addressability, and biocompatibility, nucleic acid nanostructures are extensively applied in… read more here.
Keywords: therapy based; gene; nucleic acid; gene therapy ... See more keywords
Remodeling the Inflammatory and Immunosuppressive Tumor Microenvironment for Enhancing Antiangiogenic Gene Therapy of Colorectal Cancer
Sign Up to like & getrecommendations! Published in 2024 at "Advanced Healthcare Materials"
DOI: 10.1002/adhm.202402887
Abstract: Fusobacterium nucleatum (Fn), as an intestinal pathogenic bacterium, is closely related to the occurrence, progression, and limited therapeutic efficacy of colorectal cancer (CRC). The presence of Fn within CRC communities induces an inflammatory and immunosuppressive… read more here.
Keywords: antiangiogenic gene; crc; microenvironment; inflammatory immunosuppressive ... See more keywords
Red Blood Cell Anchoring Enables Targeted Transduction and Re‐Administration of AAV‐Mediated Gene Therapy
Sign Up to like & getrecommendations! Published in 2022 at "Advanced Science"
DOI: 10.1002/advs.202201293
Abstract: Adeno‐associated virus (AAV)‐mediated gene therapy is a promising therapeutic modality for curing many diseases including monogenic diseases. However, limited tissue‐targeting and restricted re‐administration due to the vector immunogenicity largely restrict its therapeutic potential. Here, using… read more here.
Keywords: aav mediated; administration; gene therapy; transduction ... See more keywords
Ultrasound‐Assisted H2 Transmitter Enables Hydrogen‐Gene Therapy to Prevent Anesthesia/Surgery‐Induced Cognitive Impairment
Sign Up to like & getrecommendations! Published in 2025 at "Advanced Science"
DOI: 10.1002/advs.202414397
Abstract: Postoperative cognitive dysfunction (POCD) frequently occurs after surgery, resulting in extended hospitalizations, higher healthcare expenses, and potential long‐term cognitive impairment. Despite its significant impact, effective preventive and therapeutic strategies for POCD are still lacking. Neuroinflammation… read more here.
Keywords: surgery; cognitive impairment; anesthesia surgery; hydrogen gene ... See more keywords
Gene therapy decreases seizures in a model of Incontinentia pigmenti
Sign Up to like & getrecommendations! Published in 2017 at "Annals of Neurology"
DOI: 10.1002/ana.24981
Abstract: Incontinentia pigmenti (IP) is a genetic disease leading to severe neurological symptoms, such as epileptic seizures, but no specific treatment is available. IP is caused by pathogenic variants that inactivate the Nemo gene. Replacing Nemo… read more here.
Keywords: therapy decreases; gene therapy; incontinentia pigmenti; gene ... See more keywords