Gene therapy for XSCID: the first success of gene therapy

Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector.

Gene therapy·2010

Lentiviral vectors with amplified beta cell-specific gene expression.

Gene therapy·2009

Tissue-specific restriction of cyclophilin A-independent HIV-1- and SIV-derived lentiviral vectors.

Gene therapy·2008

Gene therapy for childhood immunological diseases.

Bone marrow transplantation·2007

The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors.

Gene therapy·2007

Gene therapy to inhibit xenoantibody production using lentiviral vectors in non-human primates.

Gene therapy·2006

Periviable antenatal care, labor and delivery, and initial resuscitation: Some chores to get done before you start.

Pediatric research·2026

Associations of blood and urinary heavy metals with obesity risk among children: NHANES 2015-2020.

Pediatric research·2026

Vascular access management plan (VAMP): neonatal European vascular access team-ESPR Recommendation 1.

Pediatric research·2026

The effect of listening to a mother's voice on emergence delirium in pediatric patients undergoing adenotonsillectomy: a prospective study.

Pediatric research·2026

Evidence strength of exposure-outcome associations in pediatric allergic diseases: an umbrella review of meta-analyses.

Pediatric research·2026

C-reactive protein-triglyceride glucose index and its dynamic trajectories on all-cause mortality in critically ill children: a longitudinal, retrospective cohort study.

Pediatric research·2026

Artificial Intelligence-Based Detection of ARIA on MRI During Alzheimer Disease Therapy: Expert Opinion on Responsible Clinical Integration.

AJR. American journal of roentgenology·2026

[Clinical pharmacogenetics and the use of biobanks].

Ugeskrift for laeger·2026

Low-Dose Mitomycin-C for Mucosal Overgrowth Treatment in Boston Type 1 Keratoprosthesis With Autologous Buccal Mucosa Overlay in a Patient With Severe Stevens-Johnson Syndrome.

Cornea·2026

Genomic Deletion of P-Glycoprotein from Gliomas Via the Focused Ultrasound-Delivery of a Duplex CRISPR Cas9 Ribonucleoprotein System.

Cell biomaterials·2026

Protein arginine methyltransferases in cardiovascular disease: mechanisms, therapeutic potential, and future directions.

Frontiers in physiology·2026

Target, silence, replace: a review on RNA-based drugs in modern medicine.

Frontiers in cell and developmental biology·2026

Related Experiment Videos

Gene therapy for XSCID: the first success of gene therapy

Related Concept Videos

Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector.

Lentiviral vectors with amplified beta cell-specific gene expression.

Tissue-specific restriction of cyclophilin A-independent HIV-1- and SIV-derived lentiviral vectors.

Gene therapy for childhood immunological diseases.

The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors.

Gene therapy to inhibit xenoantibody production using lentiviral vectors in non-human primates.

Periviable antenatal care, labor and delivery, and initial resuscitation: Some chores to get done before you start.

Associations of blood and urinary heavy metals with obesity risk among children: NHANES 2015-2020.

Vascular access management plan (VAMP): neonatal European vascular access team-ESPR Recommendation 1.

The effect of listening to a mother's voice on emergence delirium in pediatric patients undergoing adenotonsillectomy: a prospective study.

Evidence strength of exposure-outcome associations in pediatric allergic diseases: an umbrella review of meta-analyses.

C-reactive protein-triglyceride glucose index and its dynamic trajectories on all-cause mortality in critically ill children: a longitudinal, retrospective cohort study.

Artificial Intelligence-Based Detection of ARIA on MRI During Alzheimer Disease Therapy: Expert Opinion on Responsible Clinical Integration.

[Clinical pharmacogenetics and the use of biobanks].

Low-Dose Mitomycin-C for Mucosal Overgrowth Treatment in Boston Type 1 Keratoprosthesis With Autologous Buccal Mucosa Overlay in a Patient With Severe Stevens-Johnson Syndrome.

Genomic Deletion of P-Glycoprotein from Gliomas Via the Focused Ultrasound-Delivery of a Duplex CRISPR Cas9 Ribonucleoprotein System.

Protein arginine methyltransferases in cardiovascular disease: mechanisms, therapeutic potential, and future directions.

Target, silence, replace: a review on RNA-based drugs in modern medicine.

Related Experiment Videos

Gene therapy for XSCID: the first success of gene therapy

Related Concept Videos

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