Bench to Bedside

Where medicine, innovation, and impact intersect

[Bench to Bedside @bench2bedside_]

Looking for an robust, easy read of top clinical stories this week? Checkout our Morning Rounds...
https://t.co/8PW3uAiobQ

[Bench to Bedside @bench2bedside_]

Cas9 Nickase induces single stranded cuts into CAG repeats of R6/1 HD mice. These "nicks" generate single stranded gaps which create imperfect hairpins that are naturally "shortened" by the cellular repair mechanism itself.

Elegant safe correction of CAG repeats, at multiple disease loci, without double stranded breaks to potentially limit off-target effects.

Key findings for translation to humans:
1. Cells transduced with both viruses contracted readily, often down to <36 repeats (fig. S3, P = 0.045), suggesting that repeat sizes at least down to 47 repeats are targets for contraction.

2. CAG repeat contractions translate to shorter polyglutamine stretches (See Figure 3).

3. In vivo results show reduced mHTT inclusions, specifically in the striatum.

Limitations and additional perspective are highlighted by the Cardiff group within the paper.

@cardiffuni @UKDRI @Huntingtons @Alnylam @uniQure_NV

https://t.co/HSVqhlg92g

[Eric Vallabh Minikel @cureffi]

NN112/PRiSM, our clinical trial of divalent siRNA in prion disease, is now open to pre-symptomatic participants at genetic risk of prion disease.

https://t.co/4t60xYULPl

[Bench to Bedside @bench2bedside_]

@RNAiAnalyst We are going to have to tune in. Potentially transformative... need to dig into literature here.

[Dirk Haussecker @RNAiAnalyst]

$ARWR don't want to sound hyperbolic, but tomorrow could be one of the most important datapoints in drug development of the decade (on par with Glp1):

Can subQ-delivered RNAi substantially knock down genes in the CNS? And can we see the data of the first drug to make a real dent against Alzheimer's (safe and due to subQ could be dosed presymptomatically)?

[Bench to Bedside @bench2bedside_]

TB-500 has become popular in the peptide world, as it is sold for wound and soft-tissue healing. It is critical to understand that it is not the full thymosin Beta-4 peptide which was tested in RCTs by RegeneRx.
#TB500 #Peptides #Datafirst

[Bench to Bedside @bench2bedside_]

BPC-157 and KPV are common peptides which the FDA is considering adding to its 503a compounding list. We reviewed the literature and found the following:

#peptides #BPC157 #KPV

[Bench to Bedside @bench2bedside_]

What do you need to know about peptides? Are you feeling confused by recent news or want to brush up on your knowledge? We provide objective analyses of the current "Peptide Craze" in this weeks newsletter

https://t.co/fBzByMRN7X

[Bench to Bedside @bench2bedside_]

Ionis Pharmaceuticals had a busy last week. Catch-up on the latest data and news in our latest deep-dive. $IONS #ASOs #Alzheimers #Cardiomyopathy #Huntingtons

https://t.co/7tGbuHzzh1

[Bench to Bedside @bench2bedside_]

@EricTopol @ScienceAdvances Would be interested in comparing this data to wearables (garmin, apple watch, whoop, oura) which are heavily entrenched in the daily lives of millions.

Will see if we can do some digging...

[Eric Topol @EricTopol]

Today's sleep quality tracking relies on movement algorithms. Published today @ScienceAdvances is a wearable patch that tracks the flow of glymphatics via brain water dynamics https://t.co/98ZfsA15xf

[Bench to Bedside @bench2bedside_]

@MTSlive @cremieuxrecueil https://t.co/EnGkvBUmjM

[Bench to Bedside @bench2bedside_]

https://t.co/fYGkxKwKIM

[Bench to Bedside @bench2bedside_]

https://t.co/fYGkxKwKIM

[Bench to Bedside @bench2bedside_]

@FirstSquawk Learn more about ER-100 in our morning rounds from LAST WEEK. The ER-100 trial is recruiting patients with glaucoma and other optic neuropathies and will provide a first look at broad therapeutic potential of their platform

https://t.co/8PW3uAiobQ

[Bench to Bedside @bench2bedside_]

Very interesting application of ultrasound technology - will have to dig deeper here

https://t.co/RG4jrS70B5

[Bench to Bedside @bench2bedside_]

@CamillusMiura @MartinShkreli Planning to do a substantial deep-dive into this area. Thank you for sharing!

[Bench to Bedside @bench2bedside_]

What is Neuroplasticity? Why is Nav1.7 such an attractive therapeutic target?

Latest Morning Rounds is up!

https://t.co/KOwtVlXOmn

[Bench to Bedside @bench2bedside_]

Pain Reprocessing Therapy vs Placebo. This 2022 RCT showed the clinical impact of "re-wiring" our brain's response to chronic pain. #Nav7 #Backpain #Mindfulness #Pennmed

https://t.co/DX7VsMUv1s