Articles and Books
- Report of the 2008 ASHP Task Force on Science, AJHP
- The other side of the human genome, AJHP
- Gene therapy progress and prospects: non-viral gene therapy by systemic delivery, Gene Therapy
- Gene therapy with vascular endothelial growth factors, Biochem Soc Trans
- Progress and prospects: immune responses to viral vectors, Gene Ther
- Vectors and Delivery systems in gene therapy, Med Sci Monit
- Unity and diversity in the human adenoviruses: exploiting alternative entry pathways for gene therapy, Biochem J
- Advancing nonviral gene delivery: lipid- and surfactant-based nanoparticle design strategies, Nanomedicine [Lond]
- Gene therapy: applications to pharmacy practice, J Am Pharm Assoc
- Advances in Gene Delivery Systems, [PDF] Pharmaceut Med
- Current Advances in Retroviral Gene Therapy, [PDF] Curr Gene Ther
- Gene therapy bio-safety: scientific and regulatory issues, [PDF] Gene Ther
- Gene therapy clinical trials worldwide to 2007 – an update, [PDF] J Gene Med
- Gene therapy clinical trials worldwide 1989–2004 – an overview , J Gene Med
- Gene Therapy: The First Two Decades and the Current State-of-the-Art, [PDF] J Cell Phys
- Gene Therapy: Some History, Applications, Problems, and Prospects, Toxicol Pathol
- Progress and Prospects: Gene Therapy Clinical Trials (Part 1), Gene Ther
- Recent Advances in Nonviral Vectors for Gene Delivery, Acc Clin Res
- Gene Therapy, By Evelyn B. Kelly
- Entering the Modern Era of Gene Therapy, (Annu Rev Med)
- Applications of Gene Therapy in Cardiomyopathies, (JACC Heart Fail)
- Gene therapy: principles, challenges and use in clinical practice, (Wien Klin Wochenschr)
- Isolation, Differentiation, and Characterization of Mesenchymal Stem Cells from Human Bone Marrow, (Gastroenterol Hepatol Bed Bench)
- Early predictive factors of failure in autologous CAR T-cell manufacturing and/or efficacy in hematologic malignancies, (Blood Adv)
- Engineering the next generation of cell-based therapeutics
- TALE and TALEN genome editing technologies
- Biosafety Practices for In Vivo Viral-Mediated Gene Therapy in the Health Care Setting
- Viral-mediated gene therapy and genetically modified therapeutics: A primer on biosafety handling for the health-system pharmacist
- A call to action: Health-system pharmacists must stand up to meet the growing demand for cellular-based therapies
- Safeguards for Using Viral Vector Systems in Human Gene Therapy: A Resource for Biosafety Professionals Mitigating Risks in Health Care Settings
- Viral vector platforms within the gene therapy landscape
- Genome Engineering With Zinc-Finger Nucleases
- Current Trends and Outcomes in Cellular Therapy Activity in the United States, Including Prospective Patient-Reported Outcomes Data Collection in the Center for International Blood and Marrow Transplant Research Registry
- Handling of gene-transfer products by the National Institutes of Health Clinical Center pharmacy department
- ASHP and ASHP Foundation Pharmacy Forecast 2025: Strategic Planning Guidance for Pharmacy Departments in Hospitals and Health Systems
- Comparison of Allogeneic Stem Cell Transplant and Autologous Stem Cell Transplant in Refractory or Relapsed Peripheral T-Cell Lymphoma: A Systematic Review and Meta-analysis
- Autologous versus allogeneic mesenchymal stem cell therapy: The pros and cons
- Cell Therapy: Types, Regulation, and Clinical Benefits
- Human Gene Therapy Products Incorporating Human Genome Editing Guidance for Industry [PDF]
- A brief history of gene therapy
- Gene therapy for human genetic disease?
- Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges
- CRISPR–Cas9: A History of Its Discovery and Ethical Considerations of Its Use in Genome Editing
- Neural stem cell therapy-Brief review
- Challenges Posed by Immune Responses to AAV Vectors: Addressing Root Causes
- Management of adults and children receiving CAR T-cell therapy: 2021 best practice recommendations of the European Society for Blood and Marrow Transplantation (EBMT) and the Joint Accreditation Committee of ISCT and EBMT (JACIE) and the European Haematology Association (EHA)
- Antiemetics: ASCO Guideline Update
- Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome
- US federal oversight of biological materials and the IBC
- Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
- Hepatotoxicity in Adeno-Associated Viral Vector Gene Therapy
- Variable eculizumab clearance requires pharmacodynamic monitoring to optimize therapy for thrombotic microangiopathy after hematopoietic stem cell transplantation
- The complement system and innate immunity
- The Definition of Gene Therapy Has Changed
- Manufacturing Cell and Gene Therapies: Challenges in Clinical Translation
- ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells
- Virus treatment questioned after gene therapy death
- Advanced Therapies for Human Immunodeficiency Virus
- Clinical trial links oncolytic immunoactivation to survival in glioblastoma
- The Gene & Cell Therapy Landscape: Recent Approvals and Upcoming Therapeutics of Interest
- Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy
- Cellular therapy site-preparedness: Inpatient pharmacy implementation at a large academic medical center
- A practical approach to assess the hazardous exposure potential of investigational drugs
- Current Clinical Applications of In Vivo Gene Therapy with AAVs
- Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations
- T Cell Responses to AAV Vector Capsid Limit the Duration of Transgene Expression in Humans after Liver-Directed Gene Therapy.
- From bench to bedside: the history and progress of CAR T cell therapy
- Eculizumab precision-dosing algorithm for thrombotic microangiopathy in children and young adults undergoing HSCT
- Durability of transgene expression after rAAV gene therapy
- CD34-based enrichment of genetically engineered human T cells for clinical use results in dramatically enhanced tumor targeting
- Human Hepatocyte Transplantation: Three Decades of Clinical Experience and Future Perspective
- Chimeric Antigen Receptor T-Cell Therapy in the Outpatient Setting: An Expert Panel Opinion from the American Society for Transplantation and Cellular Therapy
- Prevention and treatment of anticipatory chemotherapy-induced nausea and vomiting in pediatric cancer patients and hematopoietic stem cell recipients: Clinical practice guideline update
- Safe Handling of BioSafety Drugs and Live Virus Vaccines
- Gene Replacement Therapy: A Primer for the Health-system Pharmacist
- Delivery of gene therapy in haemophilia treatment centres in the United States: Practical aspects of preparedness and implementation
- Mesenchymal stem cell perspective: cell biology to clinical progress
- Gene therapy for genetic diseases: challenges and future directions
- CD34—Structure, Functions and Relationship with Cancer Stem Cells
- Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
- Recent and anticipated novel drug approvals (3Q 2024 through 2Q 2025)
- Use of viruses as carriers of added genetic information
- Antisense oligonucleotides and other genetic therapies made simple
- The changing landscape of cancer cell therapies: clinical trials and real-world data
- Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies
- Chimeric antigen receptor-modified T cells: CD19 and the road beyond
- Building CRISPR Gene Therapies for the Central Nervous System: A Review
- Pfizer stops commercialization of hemophilia gene therapy Beqve
- Management of Immune-Related Adverse Events in Patients Treated With Chimeric Antigen Receptor T-Cell Therapy: ASCO Guideline
- Gene therapy: Comprehensive overview and therapeutic applications
- A new age of precision gene therapy
- 261st ENMC International Workshop: Management of safety issues arising following AAV gene therapy. 17th-19th June 2022, Hoofddorp, The Netherlands
- B-cell maturation antigen (BCMA) in multiple myeloma: rationale for targeting and current therapeutic approaches
- Best Practice Considerations by The American Society of Transplant and Cellular Therapy: Infection Prevention and Management After Chimeric Antigen Receptor T Cell Therapy for Hematological Malignancies
- Gene therapy: Practical considerations for clinical and operational pharmacy practice
- Current State of Human Gene Therapy: Approved Products and Vectors
- Immune Responses to Viral Gene Therapy Vectors
- Gene therapy vector-related myocarditis
- A perspective on oligonucleotide therapy: Approaches to patient customization
- Oral delivery of antisense oligonucleotides in man
- As new cell and gene therapies emerge from academia, we must RISE to the opportunity
- From barriers to best practices: Advancing the commercial readiness of cell and gene therapies
- Sources of Innovation in Gene Therapies - Approaches to Achieving Affordable Prices
- Adeno-associated virus as a delivery vector for gene therapy of human diseases
- Implementing standard practices in the safe handling of gene therapy and biohazardous drugs in a health-system setting
- Selecting costimulatory domains for chimeric antigen receptors: functional and clinical considerations
- Management of adults and children undergoing chimeric antigen receptor T-cell therapy: best practice recommendations of the European Society for Blood and Marrow Transplantation (EBMT) and the Joint Accreditation Committee of ISCT and EBMT (JACIE)
- The Pharmacology of Gene Therapy
- Precise genome-editing in human diseases: mechanisms, strategies and applications
- MSC based gene delivery methods and strategies improve the therapeutic efficacy of neurological diseases
Webinars and Presentations
- Gene Therapy - Is This Pharmacy’s Future? ASHP Webinar, (Handout, [PDF])
- Gene therapy: Science, History, and Implications for Pharmacy, Presented at 2007 Midyear Clinical Meeting (Handout, [PDF])
- Clinical Trials in Gene Therapy: Science, Outcomes, and Implications for Pharmacy Practice, Presented at 2008 Midyear Clinical Meeting
- Gene Therapy: Implications for Pharmacy Practice, [PDF], John Petrich, RPh, MS, Investigational Drug Service Manager, Cleveland Clinic
- Gene Therapy: Melanoma Theory to Practice, [PDF], Rowena N. Schwartz, Pharm.D., BCOP, The Johns Hopkins Hospital
- Gene Therapy for Parkinson’s Disease, [PDF], Jack J. Chen, PharmD, BCPS, CGP, Associate Professor (Neurology), Movement Disorders Center, Schools of Medicine and Pharmacy, Loma Linda University
- Results from the ASHP Gene Therapy Survey, [PDF], Susan Goodin, Pharm.D., FCCP, BCOP, Associate Director, Clinical Trials and Therapeutics, The Cancer Institute of New Jersey, Professor of Medicine, UMDNJ-Robert Wood Johnson Medical School
- Gene Therapy for Prostate Cancer, [PDF], Heidi D. Gunderson, PharmD, BCOP, Pharmacy Coordinator - Hematology/Oncology Disease Management, Instructor in Pharmacy, College of Medicine, Mayo Clinic, Rochester, Minnesota
Guidelines and Policies
- European Association of Hospital Pharmacists (EAHP) Guidance on the Pharmacy Handling of Gene Medicines, Eur J Hosp Pharm
- ASHP Formulary Management Positions-Gene Therapy, [PDF]
Other Resources
- Genetic Information, US Department of Health and Human Services
- National Human Genome Research Institute
- US Dept of Energy Human Genome Site
- Genome Hub
- Gene Therapy Journal
- Quarterly Landscape Report
Genetic Disease Information
Ethical, Legal, and Social
SNP (Single Nucleotide Polymorphisms) Information
- Human Genome Project: SNP Fact Sheet [PDF]
- National Center for Biotechnology Information
- International HapMap Project
- SNPs: Variations on a Theme
