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Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production
Optimization of recombinant adeno-associated virus (rAAV) production is essential for effective gene therapy applications....
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice
Adenosine deaminase type 2 deficiency (DADA2) is caused by bi-allelic loss-of-function mutations in ADA2. While anti-TNF t...
Systemically delivered lipid nanoparticle-mRNA encoding lysosomal acid β-glucosidase restores the enzyme deficiency in a murine Gaucher disease model
Systemically delivered lipid nanoparticle-mRNA encoding lysosomal acid β-glucosidase restores the enzyme deficiency in a murine Gaucher disease model
Gaucher disease (GD) is a rare genetically inherited illness caused by loss of lysosomal acid β-glucosidase (β-G...
hafoe: an interactive tool for the analysis of chimeric AAV libraries after random mutagenesis
hafoe: an interactive tool for the analysis of chimeric AAV libraries after random mutagenesis
Naturally occurring adeno-associated viruses (AAVs) are an integral part of gene therapy, yet engineering novel AAV varian...
Nanoparticle hydrogel system delivery of miR-494-3p to improve tendon healing by targeting CXXC4
Nanoparticle hydrogel system delivery of miR-494-3p to improve tendon healing by targeting CXXC4
Due to the poor healing capacity of tendons, the healing process is slow, with a risk of re-rupture post-injury. In this s...
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy
Transabdominal ultrasound guided AAV9-GFP delivery in fetal pigs: a translational and minimally invasive model for in utero fetal gene therapy
In utero fetal gene therapy (IUFGT) has the potential to correct severe monogenic disorders before irreversible damage occ...
hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity
hInGeTox: a human-based in vitro platform to evaluate lentivirus/host interactions that contribute to genotoxicity
Lentivirus vectors are effective for treatment of genetic disease. However, safety associated with vector related genotoxi...
High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy
High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy
The selection of an appropriate promoter is important to the design and optimisation of adeno-associated viral (AAV) vecto...
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 ...
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