Skip to content
Home
Visit our other Journals
About
Supporter
Expert Index
Editorial Board
Editorial Team
Editorial Policy
Contact
Sign up
Visit
Specialties
Hematology
Oncology
Musculoskeletal Disorder
Rare Disease
Metabolic Disorder
Cardiovascular Disease
Ophthalmology
Neurology
Infectious Disease
View all Specialties
Technologies
Cell Therapy
MSC
iPSC
Gene Therapy
Genome Editing
Extracellular Vesicles
CAR-T Therapy
TCR Therapy
Tissue-Engineering & Biomaterials
View all Technologies
Topics
Treatment
Immuno-Oncology
Trial Updates
Perspectives
Manufacturing
COVID-19
View all Topics
Conferences
ASH 2022
TCT 2022
Meeting on the Med 2022
ICLE 2022
Advanced Therapies Week 2022
TERMIS 2021
ESGCT 2021
Meeting on the Mesa 2021
Advanced Therapies 2021
View all Conferences
About
Supporter
Expert Index
Editorial Board
Editorial Team
Editorial Policy
Contact
Sign up
Rare Diseases
1:38
Overcoming manufacturing issues in gene therapies for rare diseases
John Maslowski
• 27 Jan 2022
2:12
Innovations in treating rare diseases with gene therapies
John Maslowski
• 27 Jan 2022
7:04
Applications of single-cell multi-omics analysis
Yan Zhang
• 21 Oct 2021
2:38
Expanding the repertoire of CRISPR-based genomic editors
Devyn Smith
• 29 Oct 2021
1:57
Tailored nucleases for primary hyperoxaluria
Devyn Smith
• 29 Oct 2021
1:35
Combining rAAV gene therapy with standard-of-care for the treatment of citrullinemia
Gloria Gonzalez-Aseguinolaza
• 18 May 2021
2:16
HSC gene editing highlights from ASGCT 2021
Valentina Vavassori
• 27 May 2021
2:01
Cardiosphere-derived cells in myocardial regeneration
Linda Marban
• 19 May 2021
5:31
CRISPR-Cas9 gene-edited HSPCs for Friedreich’s ataxia
Stephanie Cherqui
• 17 May 2021
1:45
Neuraminidase could improve AAV9 gene therapy for Batten disease
Kathrin Meyer
• 18 May 2021
3:41
Improving patient access to advanced therapies: are we ready for them?
Durhane Wong-Rieger
• 6 Apr 2021
1:14
The importance of specialty medical centers in delivering ATMPs
Paige Bischoff
• 11 Mar 2021
2:10
Challenges in developing gene therapies for rare diseases & CNS disorders
Kathrin Meyer
• 18 May 2021
4:30
ISCT 2021 highlights: BMT, gene therapy & lymphodepletion
Jaap Boelens
• 26 May 2021
7:49
CTNS-RD-04: HSC gene therapy for cystinosis
Stephanie Cherqui
• 17 May 2021
3:49
NextGen & GenX AAV vectors for DMD gene therapy
Arun Srivastava
• 18 May 2021
1
2
Next