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Bridging gene therapy and next-generation vaccine technologies
Bridging gene therapy and next-generation vaccine technologies
Gilbert S, Hatchett R. No one is safe until we are all safe. Sci Transl Med. 2021;13:eabl9900.Article  CAS  PubMed...
Genes for bad backs
Kang JD, Georgescu HI, McIntyre-Larkin L, Stefanovic-Racic M, Evans CH. Herniated cervical intervertebral discs spontaneou...
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration
Low back pain is the leading cause of global disability with intervertebral disc (IVD) degeneration a major cause. However...
Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis
Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis
Despite numerous studies identifying the advantages of therapies for spinal muscular atrophy (SMA), healthcare professiona...
Prime editing: therapeutic advances and mechanistic insights
Prime editing: therapeutic advances and mechanistic insights
We are often confronted with a simple question, “which gene editing technique is the best?”; the simple answer...
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent
Recombinant adeno-associated viral (AAV) vectors have emerged as prominent gene delivery vehicles for gene therapy. In the...
Placental nanoparticle-mediated IGF1 gene therapy corrects fetal growth restriction in a guinea pig model
Placental nanoparticle-mediated IGF1 gene therapy corrects fetal growth restriction in a guinea pig model
Fetal growth restriction (FGR) caused by placental insufficiency is a major contributor to neonatal morbidity and mortalit...
Unlocking patient access to gene therapy: five key practices
Unlocking patient access to gene therapy: five key practices
Zhang JX, Shugarman LR. Value-based payment and financing for cell and gene therapies: challenges and potential solutions....
Analytical characterization of full, intermediate, and empty AAV capsids
Analytical characterization of full, intermediate, and empty AAV capsids
Manufacturing of recombinant adeno-associated virus (AAV) vectors produces three types of capsids: full, intermediate, and...
Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate
Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate
Neospora caninum is an obligate intracellular protozoan that affects several animal species. It is not pathogenic for huma...
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice
Adeno-associated viruses (AAV) are commonly used in the scientific field due to their diverse application range. However, ...
Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Adoptive cell therapy (ACT), particularly chimeric antigen receptor (CAR)-T cell therapy, has emerged as a promising appro...
Precision ophthalmology: a call for Africa not to be left in the dark
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. Efficacy and safety of voretigene neparvovec (AAV2-hR...
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a
Efficient manufacturing of recombinant Adeno-Associated Viral (rAAV) vectors to meet rising clinical demand remains a majo...
A multinational survey of potential participant perspectives on ocular gene therapy
A multinational survey of potential participant perspectives on ocular gene therapy
Amidst rapid advancements in ocular gene therapy, understanding patient perspectives is crucial for shaping future treatme...
Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
Glial cell line-derived neurotrophic factor (GDNF) protects dopaminergic neurons in various models of Parkinson’s di...
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
Small promoters capable of driving potent neuron-restricted gene expression are required to support successful brain circu...
Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa
Somatic gene therapy will be one of the most exciting practices of genetic medicine in Africa and is primed to offer a ...
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
Glutaric Aciduria type I (GA1) is a rare neurometabolic disorder caused by mutations in the GDCH gene encoding for glutary...
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
To the best of our knowledge, this effort represents the most comprehensive attempt to understand and develop solutions to...
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
Gene therapy offers a potential alternative to the surgical treatment of epilepsy, which affects millions of people and is...
Implications of maternal-fetal health on perinatal stem cell banking
Implications of maternal-fetal health on perinatal stem cell banking
Cell based therapies are being assessed for their therapeutic potential across a variety of diseases. Gestational tissues ...
A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies
A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies
In the rapidly evolving landscape of biotechnologies, cell and gene therapies are being developed and adopted at an unprec...
Cost of gene therapy
Since its earliest days and during the ensuing 5–6 decades, the field of human gene therapy has been char...
Approval and therapeutic value of gene therapies in the US and Europe
Gene therapies are a fast-growing area of innovation and hold promise for the treatment of many diseases currently with un...
Access to affordable medicines: obligations of universities and academic medical centers
The authors thank Katharine Gleason, MPH and Mariah Johnson, MPH for research assistance and administrative support. The a...
Successes and challenges in clinical gene therapy
Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provid...
The estimated annual financial impact of gene therapy in the United States
The estimated annual financial impact of gene therapy in the United States
Gene therapy is a new class of medical treatment that alters part of a patient’s genome through the replacement, del...
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
Adeno-associated virus (AAV) vector gene therapy is a promising approach to treat rare genetic diseases; however, an ongoi...
p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma
p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma
Dry gene powder is a novel non-viral gene-delivery system, which is inhalable with high gene expression. Previously, we sh...
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