The Vector

Volume 10, Issue 6: June 2021


Editorial Team

Edith Pfister, Ph.D. – Editor, The Vector
Karen Bulaklak, Ph.D. – Associate Editor, The Vector
Jon Brudvig, Ph.D. – Junior Editor, The Vector

Inside This Issue

Leadership Message
Breaking Through
From Molecular Therapy
Society News
Career Center
Public Policy
Industry News

Leadership Message


Kicking Off the 2021-2022 Programming Year

Hello ASGCT Members,

Each year in the wake of the Annual Meeting the entire staff team and I typically take a collective moment to reflect on the past program year, reviewing the Society’s successes and advances in the field while preparing for the new initiatives right around the corner. The last year has been like no other and I want to sincerely thank each of the more than 500 volunteers who served on a standing, scientific, or abstract review committee, all of whom helped make the 2020-2021 program year possible. Thank you also to Drs. Stephen Russell, Jennifer Adair, Stephen Hart, and Richard Morgan, whose terms on the Board of Directors ended this month, for your steadfast commitment to the Society and the entire field of gene and cell therapy.

Looking ahead, we are leaping right into the 2021-2022 programming year, opening applications for six Career Development Awards and three Diversity and Inclusion Awards, including two new fellowships.  The Society is also hosting two free virtual events this month that I encourage you to attend.

Tomorrow, June 11, we’re holding  a seminar on Academic Writing Dos and Don’ts that will focus on both traditional and non-traditional forms of professional scientific writing. This event is the second in our series of monthly Professional Development Seminars, and information on future seminars will be posted as it gets confirmed. On June 22, join us for another free event, the Forum on Gene Therapy for Underserved Populations. Speakers will explore new approaches to developing gene therapies for people with ultra-rare diseases and people in lower-income countries. Our new ASGCT President, Beverly Davidson, Ph.D., has made it a goal to work with ASGCT’s committees and staff to create more events like these, so stay tuned!

I want to remind everyone that you can watch all the content from the 24th Annual Meeting on demand (as long as you’re registered) on the meeting platform and on ASGCT.org through August 13. I was truly amazed at all the fantastic research that was presented at the meeting and I hope you’ll take the time to go back and watch anything you missed. The Annual Meeting is always a testament to how far the field has come, and I certainly came away from it feeling excited for the year ahead.   

 

Sincerely,

David Barrett, JD
ASGCT CEO

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Breaking Through


Restoring Neuronal Chloride Homeostasis with Anti-NKCC1 Gene Therapy Rescues Cognitive Deficits in a Mouse Model of Down Syndrome

Parrini M, Naskar S, Alberti M, Colombi I, Morelli G, Rocchi A, Nanni M, Piccardi F, Charles S, Ronzitti G, Mingozzi F, Contestabile A, Cancedda L

DOI: https://doi.org/10.1016/j.ymthe.2021.05.023

Summary by Edith Pfister, Ph.D.

Alterations in chloride homeostasis caused by changes in activity of the chloride importer NKCC1 or the exporter KCC2 occur in a variety of brain diseases. The NKCC1 transporter is increased in samples from patients with Down syndrome and the transporter is upregulated in the Ts65Dn mouse model. In a new study, Parrini et al. develop a gene therapy strategy for targeting NKCC1. First, the authors designed and screened artificial micro RNAs (amiR) targeting the NKCC1 mRNA in cultured neurons. The amiR lowered NKCC1 and decreased chloride in primary cortical neurons. In hippocampal neurons, knockdown of NKCC1 rescued defects in GABAergic signaling in neurons from Ts65Dn mice. Although responses to GABA signaling are heterogeneous in both WT and Ts65Dn neurons, knockdown of NKCC1 restored network-wide inhibitory signaling by GABA.

In hippocampal slices, expression of the amiR targeting NKCC1 in neurons in the hippocampus of Ts65Dn mice reduced levels of NKCC1 and decreased spontaneous firing activity to levels comparable to WT suggesting that GABAAR-mediated inhibition was restored. In vivo expression of the amiR also rescued behavior in the contextual fear conditioning, novel object, and object location tests, which evaluate associative, long-term, and spatial memory. Changes were still present five to six months post injection of the amiR.

In addition to Down syndrome, disruptions in chloride homeostasis are also present in a variety of other brain diseases. Targeting NKCC1 could represent a potential gene therapy approach to multiple diseases. This study provides proof of concept for this approach.

From Molecular Therapy


This month, read these new issues of Molecular Therapy family journals:
Molecular Therapy
Molecular Therapy: Nucleic Acids

You can still submit your papers for a special issue of Molecular Therapy: Oncolytics on combination gene and cellular immunotherapies. These immunotherapies pose exciting regulatory challenges to the field and we’ll highlight preclinical, clinical, and regulatory advances in this special issue. More information on topics and article types is available here. Submit your paper by August 1.

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Society News


Read #ASGCT21 Blog Coverage and Watch Sessions On Demand

If you missed any sessions from the 24th Annual Meeting, catch up by reading our committee members' coverage on the blog:

Registered attendees can also watch all the sessions from the meeting on demand on the meeting platform and on ASGCT.org through August 13.

Register for Free Events and Update Your Profile 

Tomorrow, June 11, is our second monthly Professional Development Seminar, Academic Writing Dos and Don'ts. During the seminar, speakers will discuss the challenges and best practices professionals face while communicating with various populations in various platforms. Additionally, a panel of Molecular Therapy editors will be taking your questions. 

On June 22, attend the Forum on Gene Therapy for Underserved Populations. Speakers from leading efforts in this space will identify the scope of development; discuss innovative approaches and business models; and spark conversation to advance these treatments for all who may benefit from them. 

Finally, as we progress into Pride Month, we want to remind you to fill out the demographics section of your profile! Let us know more about you, including your gender identity, pronoun preference, race/ethnicity, and more, so we can understand and serve you better as a member of ASGCT.

Now Open: Career Development, Diversity & Inclusion Awards

We're into award season, and ASGCT members can now apply for two types of funded awards totalling nearly $1 million! The Career Development Awards support six members working toward independence in their gene and cell therapy careers, and in 2021 the values have doubled to $100,000 each! We also have new Diversity & Inclusion Awards that will fund two fellowships for underrepresented minorities and one opportunity for a member to research a condition disproportionately affecting minorities. Learn more about all the awards here.

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Career Center


Are you looking for a job in the field of gene and cell therapy? Check out the new ASGCT Career Center for great opportunities with industry, government, and academic organizations. Sign up to receive alerts for open jobs in your area.

If you're from a recruiting institution, advertise in the Featured Jobs section to target the 4,000+ audience of The Vector.

Featured Jobs

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Public Policy


Law Passed With ASGCT Provision to Further Regenerative Medicine 

The TRANSPLANT Act (HR 941), containing an ASGCT-recommended provision, was signed into law by President Biden last month. The provision codifies the furthering of regenerative medicine into the Public Health Service Act, and explicitly includes gene and genetically modified cell therapies in the definition of regenerative medicine. The Society views this calling of NIH attention to regenerative medicine to be a step toward ensuring continued federal research funding to the field that was initiated, and provided through 2020, by the 21st Century Cures Act. The provision is within the law that reauthorizes for another five years the C.W. Bill Young Cell Transplantation Program, the donor matching program for patients in need of a bone marrow, blood stem cell, or cord blood transplant. 

Society Supports Access to Genetic Testing

ASGCT signed on last month to a letter to Congressmen Eric Swalwell (D-CA) and Scott Peters (D-CA) in support of legislation that would require state Medicaid programs to cover genomic sequencing tests. Specifically, the letter, led by The Assistance Fund, requests coverage of various diagnostic sequencing technologies (including whole genome sequencing, whole exome sequencing, and gene panels) to allow access to whichever type of diagnostic test a clinician deems necessary to accurately diagnose individual patients. The two representatives are working on a compromise bill that will reconcile differences between their two bills. Increasing consistent approaches to Medicaid coverage across states could improve timely diagnosis, and therefore treatment, of genetic diseases. 

For more policy news, sign up to receive The Advocate every month!

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Industry News


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2021

ASGCT Policy Summit

September 22-24, 2021

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