Featured
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Review Article
| Open AccessLooking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa
- Nchangwi Syntia Munung
- , Obiageli E. Nnodu
- & Ambroise Wonkam
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Article
| Open AccessSuccesses and challenges in clinical gene therapy
- Donald B. Kohn
- , Yvonne Y. Chen
- & Melissa J. Spencer
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Article
| Open Access
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
- Alexander Kistner
- , Jessica A. Chichester
- & Barbara A. Sullivan
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Perspective |
Equitable access to cell and gene therapies in South Africa: opportunities and hurdles
- Candice Laverne Hendricks
- , Marco Alessandrini
- & Michael Sean Pepper
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Article |
Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells
- Young-In Kim-Hoehamer
- , Janice M. Riberdy
- & Sheng Zhou
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Review Article
| Open Access
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)
- Jennifer E. Adair
- , Lindsay Androski
- & Boro Dropulić
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Article
| Open Access
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer
- Krystal Sandza
- , Annie Clark
- & Christian Vettermann
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Review Article
| Open Access
Retroviral gene therapy in Germany with a view on previous experience and future perspectives
- Michael A. Morgan
- , Melanie Galla
- & Axel Schambach
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Review Article |
An overview of development in gene therapeutics in China
- Dawei Wang
- , Kang Wang
- & Yujia Cai
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Article |
Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells
- Fatima Rodriguez-Fornes
- , Oscar Quintana-Bustamante
- & Guillermo Guenechea
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Original Article
| Open Access
Strategy to detect pre-existing immunity to AAV gene therapy
- L Falese
- , K Sandza
- & S Fong
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Short Communication |
Long-term correction of hemophilia A mice following lentiviral mediated delivery of an optimized canine factor VIII gene
- J M Staber
- , M J Pollpeter
- & P B McCray
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Original Article |
Delivery of human erythropoietin gene with an adeno-associated virus vector through parotid glands to treat renal anaemia in a swine model
- C Ma
- , Z Fan
- & Z Shan
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Review |
The state of gene therapy research in Africa, its significance and implications for the future
- P Arbuthnot
- , M B Maepa
- & M S Pepper
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Original Article |
Evidence for the in vivo safety of insulated foamy viral vectors
- D L Browning
- , E M Everson
- & G D Trobridge
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Original Article |
Direct interaction of human serum proteins with AAV virions to enhance AAV transduction: immediate impact on clinical applications
- M Wang
- , J Sun
- & C Li
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Original Article |
siRNA targeting the κ light chain constant region: preclinical testing of an approach to nonfibrillar and fibrillar light chain deposition diseases
- X Ma
- , P Zhou
- & R L Comenzo
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Short Communication |
Excision of HIV-1 DNA by gene editing: a proof-of-concept in vivo study
- R Kaminski
- , R Bella
- & K Khalili
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Original Article |
Evaluation of the biological differences of canine and human factor VIII in gene delivery: implications in human hemophilia treatment
- Q Wang
- , B Dong
- & W Xiao
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Original Article
| Open Access
Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice
- C D Anderson
- , S Moisyadi
- & R V Shohet
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Short Communication
| Open Access
Measurement of lentiviral vector titre and copy number by cross-species duplex quantitative PCR
- I Christodoulou
- , P Patsali
- & C W Lederer
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Original Article |
Downregulation of HO-1 promoted apoptosis induced by decitabine via increasing p15INK4B promoter demethylation in myelodysplastic syndrome
- D Ma
- , Q Fang
- & J S Wang
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Original Article |
Novel electric power-driven hydrodynamic injection system for gene delivery: safety and efficacy of human factor IX delivery in rats
- T Yokoo
- , K Kamimura
- & Y Aoyagi
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Original Article |
Preclinical development of siRNA therapeutics for AL amyloidosis
- B M Hovey
- , J E Ward
- & D C Seldin
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Original Article |
Long-term phenotypic correction in factor IX knockout mice by using phiC31 integrase-mediated gene therapy
- A Keravala
- , C L Chavez
- & M P Calos
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Enabling Technologies |
Signed outside: a surface marker system for transgenic cytoplasmic proteins
- V Wohlgensinger
- , R Seger
- & U Siler
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia
Gene therapy of hematological disorders: current challenges