Reviews

• Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency

• Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases

• Gene therapy then and now: A look back at changes in the field over the past 25 years

• Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases

• Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders

• Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer

• Gene therapy for alpha-1 antitrypsin deficiency: an update

• Defining Scholarship for Today and Tomorrow

• Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

• Gene Therapy for Rare Neurological Disorders

• CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing

• The rapidly evolving state of gene therapy

• CAR T-Cell Therapy: Progress and Prospects

• Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans

• Training the next generation of biomedical investigators in glycosciences

• Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods

• Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency

• Current status of gene therapy for α-1 antitrypsin deficiency

• Gene-based therapy for alpha-1 antitrypsin deficiency

• Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome

• Gene therapy for alpha-1 antitrypsin deficiency

• Endocrine parameters of cystic fibrosis: back to basics

• Gene therapy for cystic fibrosis

• Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies

• The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency

• Gene therapy: the first two decades and the current state-of-the-art

• Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis

• Recurrent pneumonia in children: a case report and approach to diagnosis

• Adeno-associated virus-based gene therapy for inherited disorders

• Recent developments in recombinant AAV-mediated gene therapy for lung diseases

• Adeno-associated virus-mediated gene transfer for lung diseases

• Adeno-associated virus: a ubiquitous commensal of mammals

• Recombinant adeno-associated virus vectors for gene therapy

• The pyruvate dehydrogenase complex as a target for gene therapy

• Production of clinical-grade recombinant adeno-associated virus vectors

• Virus-based gene delivery systems