Clinical experiences with systemically administered siRNA-based therapeutics in cancer

Related Experiment Video

Updated: Mar 30, 2026

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
09:09

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Published on: May 2, 2019

8.1K

Jonathan E Zuckerman1, Mark E Davis2

  • 1Department of Pathology and Laboratory Medicine, University of California Los Angeles, Los Angeles, California 90095, USA.

Nature Reviews. Drug Discovery
|November 17, 2015
PubMed

Insights

Small interfering RNA (siRNA) therapies show promise for treating solid tumors. Early clinical trials demonstrate safety and efficacy in inhibiting gene targets, providing valuable insights for future cancer treatment development.

Area of Science:

  • Oncology
  • Molecular Biology
  • Pharmacology

Background:

  • Small interfering RNA (siRNA)-based therapies represent a novel approach in cancer treatment.
  • Early-stage clinical trials have been conducted for patients with solid tumors.

Purpose of the Study:

  • To critically assess Phase I clinical studies of siRNA-based anticancer therapies.
  • To discuss implications for future clinical trial design and development.

Main Methods:

  • Review of completed Phase I clinical trials involving siRNA therapeutics for solid tumors.
  • Analysis of safety, efficacy, dosage, and scheduling from trial data.

Main Results:

  • Phase I trials indicate that siRNA therapeutics can safely and effectively inhibit targeted gene products in cancer patients.
  • Pioneering studies have provided crucial information on appropriate dosing and treatment schedules.

Conclusions:

  • siRNA-based therapies are a promising anticancer strategy with demonstrated safety and efficacy.
  • Lessons learned from early trials are vital for optimizing future clinical development and overcoming challenges in siRNA therapeutics.

More Related Videos

Porous Silicon Microparticles for Delivery of siRNA Therapeutics08:31

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

Published on: January 15, 2015

11.6K
DNA Vector-based RNA Interference to Study Gene Function in Cancer13:10

DNA Vector-based RNA Interference to Study Gene Function in Cancer

Published on: June 4, 2012

21.1K

Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
19.0K
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
9.1K
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

1.8K
Experimental RNAi02:15

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
8.3K
Treatment Resistent Cancers02:56

Treatment Resistent Cancers

1.5K
Treatment Resistant Cancers02:56

Treatment Resistant Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
3.9K