Rare Diseases

Overcoming manufacturing issues in gene therapies for rare diseases 1:38
Overcoming manufacturing issues in gene therapies for rare diseases
John Maslowski • 27 Jan 2022
Innovations in treating rare diseases with gene therapies 2:12
Innovations in treating rare diseases with gene therapies
John Maslowski • 27 Jan 2022
Applications of single-cell multi-omics analysis 7:04
Applications of single-cell multi-omics analysis
Yan Zhang • 21 Oct 2021
Expanding the repertoire of CRISPR-based genomic editors 2:38
Expanding the repertoire of CRISPR-based genomic editors
Devyn Smith • 29 Oct 2021
Tailored nucleases for primary hyperoxaluria 1:57
Tailored nucleases for primary hyperoxaluria
Devyn Smith • 29 Oct 2021
Combining rAAV gene therapy with standard-of-care for the treatment of citrullinemia 1:35
Combining rAAV gene therapy with standard-of-care for the treatment of citrullinemia
Gloria Gonzalez-Aseguinolaza • 18 May 2021
HSC gene editing highlights from ASGCT 2021 2:16
HSC gene editing highlights from ASGCT 2021
Valentina Vavassori • 27 May 2021
Cardiosphere-derived cells in myocardial regeneration 2:01
Cardiosphere-derived cells in myocardial regeneration
Linda Marban • 19 May 2021
CRISPR-Cas9 gene-edited HSPCs for Friedreich’s ataxia 5:31
CRISPR-Cas9 gene-edited HSPCs for Friedreich’s ataxia
Stephanie Cherqui • 17 May 2021
Neuraminidase could improve AAV9 gene therapy for Batten disease 1:45
Neuraminidase could improve AAV9 gene therapy for Batten disease
Kathrin Meyer • 18 May 2021
Improving patient access to advanced therapies: are we ready for them? 3:41
Improving patient access to advanced therapies: are we ready for them?
Durhane Wong-Rieger • 6 Apr 2021
The importance of specialty medical centers in delivering ATMPs 1:14
The importance of specialty medical centers in delivering ATMPs
Paige Bischoff • 11 Mar 2021
Challenges in developing gene therapies for rare diseases & CNS disorders 2:10
Challenges in developing gene therapies for rare diseases & CNS disorders
Kathrin Meyer • 18 May 2021
ISCT 2021 highlights: BMT, gene therapy & lymphodepletion 4:30
ISCT 2021 highlights: BMT, gene therapy & lymphodepletion
Jaap Boelens • 26 May 2021
CTNS-RD-04: HSC gene therapy for cystinosis 7:49
CTNS-RD-04: HSC gene therapy for cystinosis
Stephanie Cherqui • 17 May 2021
NextGen & GenX AAV vectors for DMD gene therapy 3:49
NextGen & GenX AAV vectors for DMD gene therapy
Arun Srivastava • 18 May 2021
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