With Policy Summit early registration rates ending today, August 23, now is the perfect time to secure your spot at the best price! asgct.org/policysummit #ASGCTAdvocacy

Engineered minimal type I CRISPR-Cas system for transcriptional activation and base editing in human cells nature.com/articles/s4146…

The therapeutic implications of all-in-one AAV-delivered epigenome-editing platform in neurodegenerative disorders nature.com/articles/s4146…

Genome editing with DNA-dependent polymerases nature.com/articles/s4158…

Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models nature.com/articles/s4143…

James McPartland is part of a team that is using a new gene editing platform, and as proof-of-concept, are targeting two diseases: Angelman syndrome (AS) and H1-4 syndrome (HIST1H1E syndrome). #CoIMonday scge.mcw.edu/phase-2-platfo…

Birth of protein folds and functions in the virome nature.com/articles/s4158…

Artificial intelligence in medical genomics nature.com/articles/s1003…

Do you have a colleague in mind who is (or are you) significantly contributing to the #CGT field? Nominate them for one of 10 ASGCT Annual Meeting Awards through Sept. 16! Criteria + descriptions on our website. bit.ly/3WT048l #ASGCT2025

Calling all clinicians: Expand your knowledge of #genetherapy + #celltherapy while earning CME! This FREE opportunity is provided by Partners for Advancing Clinical Education and PlatformQ Health in collaboration with ASGCT and NORD. Register for free + start learning more! bit.ly/3T14Kqj

ASGCT briefed the @HouseBudgetGOP on enabling access to transformative cell & gene therapies. We shared an in-depth look at the #CGT pipeline, manufacturing hurdles, and policy solutions to improve patient accessibility. #ASGCTAdvocacy Learn more: asgct.org/advocacy

Register for a Sept. 20 in-person event at Children's Hospital, The Safe and Effective Practice of AAV #GeneTherapy! During this full-day seminar, you'll hear from ASGCT Board Member Lindsey A. George MD, Past President Beverly Davidson, Denise Sabatino, + more. bit.ly/4g35xkG

SCGE researchers reviewed AAV biology with an emphasis on current vector engineering strategies and manufacturing technologies and discussed how rAAVs are being employed in ongoing clinical trials for various diseases. #PublicationFriday nature.com/articles/s4139…

FDA invites applications for the FDA Rare Neurodegenerative Disease Grant Program to help address unmet needs in rare neurodegenerative diseases for children & adults. Apply by Oct. 22. fda.gov/industry/medic…

CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model nature.com/articles/s4143…

Check out new research partly supported by NIH Common Fund ’s Somatic Cell Genome Editing (#SCGE) program to find out how #GeneEditing can potentially lead to treatments for inherited hearing loss! NCATS co-leads Somatic Cell Genome Editing (SCGE) Outreach along with NINDS

The cost of #healthcare is heavier on #RareDisease patients, particularly those in marginalized groups, according to our joint survey with @RareDiseaseDiv1. This challenge requires all of us to fix it: providers, insurers, legislators, advocates. Read more:

Check out this study, "Inequities in the Rare Disease Community", from the RDDC and NORD (NORD). How do you work to combat inequities in your rare disease research? #raredisease #healthequity #healthcarecosts #sdoh rarediseases.org/wp-content/upl…

The next ELSI (ELSIhub | CERA) Friday Forum is on 9/13 at 12pm ET. This forum is titled "Enhancing Access for Historically Marginalized Populations Using the Translational Genomics Pipeline". Find more information at elsihub.org/ELSIFridayForum

SCGE researchers found that AAV2 viral capsids exhibit strong capacity as an endogenous CEST contrast agent and can potentially be used for monitoring and evaluation of AAV vector-mediated gene therapy protocols. #PublicationFriday pubmed.ncbi.nlm.nih.gov/38651648/